On October 21, 2025 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported that a global licensing and collaboration agreement with Novartis, previously announced on September 2, 2025, 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.

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Summary of License and Collaboration Agreement

Novartis received an exclusive worldwide license to research, develop, manufacture, and commercialize ARO-SNCA, a preclinical stage program that utilizes Arrowhead’s Targeted RNAi Molecule (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 may also 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

Arrowhead will receive a $200 million upfront payment from Novartis and 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.

(Press release, Arrowhead Pharmaceuticals, OCT 21, 2025, View Source [SID1234656875])

On October 21, 2025 Xspray pharma reported that the U.S. Food and Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) for XS003 (nilotinib) for review under the 505(b)(2) regulatory pathway. The FDA has set a PDUFA date to June 18, 2026, which is the date by when the agency is expected to announce a decision on the application.

XS003 is an improved formulation of nilotinib (Tasigna) for the treatment of chronic myeloid leukemia (CML), developed using Xspray’s proprietary HyNap technology. Data demonstrate bioequivalence to the reference product at less than half the dose, as well as a significantly reduced food effect (29% compared to 82% for Tasigna). The food effect associated with the reference product complicates patient adherence and is related to the requirement that the patient must be in a fasting state, which is included in its so-called "boxed warning" regarding QTc prolongation and the risk of sudden death. The significantly reduced food effect with XS003 means it is expected that it may be taken with or without food and thereby avoid warning text regarding food interaction. Any final labeling and related warnings will be determined by the FDA during the review process.

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"The FDA’s decision to accept our NDA for review marks an important milestone," says Per Andersson, CEO of Xspray Pharma. "With two product candidates under FDA review, we are demonstrating that the HyNap platform has broad applicability and the potential to deliver more improved TKIs to the market."

Together with the company’s first product candidate Dasynoc, XS003 addresses a U.S. market of approximately USD 2.7 billion. The FDA’s notification confirms that the application is complete and that a full review is now underway. XS003 is manufactured at the same external facility as Dasynoc.

"Since the FDA now has a solid understanding of our manufacturing process, I am confident that the upcoming Pre-Approval Inspection will be completed without observations. It is essentially the same process that has previously been inspected. Furthermore, we are working closely with the manufacturer to ensure that all planned improvements to the other parts of the facility that previously received observations are completed well ahead of the PDUFA date," comments CEO Per Andersson.

(Press release, Xspray, OCT 21, 2025, View Source [SID1234656877])

On October 21, 2025 SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany, reported that long-term efficacy and safety data from the Phase 3 DeFi trial of OGSIVEO (nirogacestat), an oral gamma secretase inhibitor for the treatment of adults with progressing desmoid tumors who require systemic treatment, were published in the Journal of Clinical Oncology (JCO). The long-term follow-up data from DeFi, which was a global, randomized, multicenter, double-blind, placebo-controlled trial, were previously presented at the 2024 Connective Tissue Oncology Society Meeting. The new results published in JCO utilized a December 2024 data cutoff date (the final data cut of the clinical trial) and showed that longer-term treatment with OGSIVEO was associated with further reductions in tumor size, an increase in objective response rate (ORR) with additional partial responses (PRs) and complete responses (CRs), sustained improvement in patient reported outcomes, and a consistent safety profile compared to the April 2022 data cut off utilized for the primary analysis of the trial. The JCO e-publication can be accessed at the following link: View Source

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"Desmoid tumors are locally aggressive and complex tumors whose unpredictable growth can cause significant pain, functional impairment, and emotional distress. For many patients, these tumors disrupt daily life in ways that are often underestimated, so advancing treatment options that offer durable symptom relief and tumor control can make a meaningful difference for patients," said Ravin Ratan, M.D., M.Ed., Associate Professor, Department of Sarcoma Medical Oncology, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center in Houston and lead author of the JCO publication. "While the optimal duration of therapy may vary for many patients and is best decided between individual patients and their physicians, the new data published in the JCO provide physicians with additional information regarding the long-term safety and efficacy of nirogacestat, and will help inform treatment decisions and improve patient care."

In the Phase 3 DeFi trial primary analysis, which was previously published in the New England Journal of Medicine, OGSIVEO showed significant improvement versus placebo in progression-free survival (PFS), objective response rate (ORR), and patient-reported outcomes (PRO) in adult patients with progressing desmoid tumors (DT; median [range] exposure: 20.6 [0.3-33.6] months).1 In the JCO publication, long-term efficacy and safety were evaluated in patients randomized to OGSIVEO and followed through the final data-cutoff date of December 2024. The median duration of OGSIVEO treatment in these patients was 33.6 (0.3 to 61.8) months.

Objective response rates (ORR) improved with long-term OGSIVEO treatment. While ORR was 34.3% (n = 24) in year 1, it increased to 45.7% (n = 32) in patients who received OGSIVEO for up to 4 years, with three additional complete responses (CRs) and three additional partial responses (PRs) since the primary analysis and yielding 24 (34.3%) PRs and 8 (11.4%) CRs in total. The median best percent reduction from baseline in target tumor size by RECIST 1.1 with continuous OGSIVEO treatment was −32.3% at year one (n=46) and −75.8% (n=15) for patients completing at least four years of treatment. Improvements in patient-reported outcomes (PROs) of pain, desmoid tumor-specific symptom severity, desmoid tumor-specific physical functioning, global health status/quality of life and role functioning occurred early (as early as Cycle 2, the first post-treatment timepoint evaluated as disclosed at the primary analysis) and were sustained with up to 45 months of treatment with OGSIVEO.

Overall, the incidence and severity of frequently reported treatment emergent adverse events (TEAEs) decreased through years two, three and four of treatment. The most common adverse events (>15%) reported in patients receiving OGSIVEO were diarrhea, ovarian toxicity, rash, nausea, fatigue, stomatitis, headache, abdominal pain, cough, alopecia, upper respiratory tract infection, and dyspnea. Please see Important Safety Information below, including Warnings & Precautions relating to diarrhea, ovarian toxicity, hepatotoxicity, non-melanoma skin cancers, electrolyte abnormalities, and embryo-fetal toxicity.2

"We are pleased that long-term continuous nirogacestat treatment for up to four years was associated with additional late responses and further tumor size reductions," said Uche Iloeje, M.D., Senior Vice President, Global Head of Medical Affairs at SpringWorks Therapeutics. "These data represent the largest prospective analysis from a randomized controlled clinical trial of long-term exposure to any systemic agent for desmoid tumors and provide valuable insights on the benefits of OGSIVEO for patients with desmoid tumors."

About the DeFi Trial

DeFi (NCT03785964) was a global, randomized (1:1), multicenter, double-blind, placebo-controlled pivotal Phase 3 trial that evaluated the efficacy, safety and tolerability of nirogacestat in adult patients with progressing desmoid tumors. The double-blind phase of the study randomized 142 patients (nirogacestat, n=70; placebo n=72) to receive 150 mg of nirogacestat or placebo twice daily. Key eligibility criteria included tumor progression by ≥20% as measured by Response Evaluation Criteria in Solid Tumors (RECIST 1.1) within 12 months prior to screening. The primary endpoint was progression-free survival, as assessed by blinded independent central review, or death by any cause. Secondary and exploratory endpoints included safety and tolerability measures, objective response rate, duration of response, changes in tumor volume assessed by magnetic resonance imaging (MRI), and changes in patient-reported outcomes. DeFi also included an open-label extension phase.

About Desmoid Tumors

Desmoid tumors are rare, locally aggressive tumors of the soft tissues that can be serious, debilitating, and, in rare cases when vital structures are impacted, life-threatening.3,4

Desmoid tumors are most commonly diagnosed in patients between the ages of 20 and 44 years, with a two-to-three times higher prevalence in females.5,6 In the U.S., up to 1650 people are diagnosed with desmoid tumors every year.5,7,8

Although desmoid tumors do not metastasize, they can be associated with recurrence rates of up to 77% after surgical resection.6,9 Desmoid tumor experts and treatment guidelines now recommend systemic therapies as first-line intervention for most tumor locations requiring treatment.10,11

About OGSIVEO (nirogacestat)

OGSIVEO (nirogacestat) is an oral, selective, small molecule gamma secretase inhibitor approved in the United States and European Union as monotherapy for the treatment of adult patients with progressing desmoid tumors who require systemic treatment.

The FDA and the EMA have granted Orphan Drug designation for OGSIVEO for the treatment of desmoid tumors.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Diarrhea: Diarrhea occurred in 84% of patients treated with OGSIVEO. Grade 3 events occurred in 16% of patients. Monitor patients and manage using antidiarrheal medications. Modify dose as recommended.
Ovarian Toxicity: Female reproductive function and fertility may be impaired in patients treated with OGSIVEO. Impact on fertility may depend on factors like duration of therapy and state of gonadal function at time of treatment. Long-term effects on fertility have not been established. Advise patients on the potential risks for ovarian toxicity before initiating treatment. Monitor patients for changes in menstrual cycle regularity or the development of symptoms of estrogen deficiency, including hot flashes, night sweats, and vaginal dryness.
Hepatotoxicity: ALT or AST elevations occurred in 30% and 33% of patients, respectively. Grade 3 ALT or AST elevations (>5 × ULN) occurred in 6% and 2.9% of patients. Monitor liver function tests regularly and modify dose as recommended.
Non-Melanoma Skin Cancers: New cutaneous squamous cell carcinoma and basal cell carcinoma occurred in 2.9% and 1.4% of patients, respectively. Perform dermatologic evaluations prior to initiation of OGSIVEO and routinely during treatment.
Electrolyte Abnormalities: Decreased phosphate (65%) and potassium (22%) occurred in OGSIVEO-treated patients. Phosphate <2 mg/dL occurred in 20% of patients. Grade 3 decreased potassium occurred in 1.4% of patients. Monitor phosphate and potassium levels regularly and supplement as necessary. Modify dose as recommended.
Embryo-Fetal Toxicity: Oral administration of nirogacestat to pregnant rats during the period of organogenesis resulted in embryo-fetal toxicity at maternal exposures below human exposure at the recommended dose of 150 mg twice daily. Advise pregnant women of the potential risk to a fetus. Advise females and males of reproductive potential to use effective contraception during treatment with OGSIVEO and for 1 week after the last dose.
ADVERSE REACTIONS

The most common (≥15%) adverse reactions were diarrhea, ovarian toxicity, rash, nausea, fatigue, stomatitis, headache, abdominal pain, cough, alopecia, upper respiratory tract infection, and dyspnea.
Serious adverse reactions occurring in ≥2% of patients were ovarian toxicity (4%).
The most common laboratory abnormalities (≥15%) were decreased phosphate, increased urine glucose, increased urine protein, increased AST, increased ALT, and decreased potassium.
DRUG INTERACTIONS

CYP3A Inhibitors and Inducers: Avoid concomitant use with strong or moderate CYP3A inhibitors (including grapefruit products, Seville oranges, and starfruit) and strong or moderate CYP3A inducers.
Gastric Acid Reducing Agents: Avoid concomitant use with proton pump inhibitors and H2 blockers. If concomitant use cannot be avoided, OGSIVEO can be staggered with antacids (e.g., administer OGSIVEO 2 hours before or 2 hours after antacid use).
Consult the full Prescribing Information prior to and during treatment for important drug interactions.
To report suspected adverse reactions, contact SpringWorks Therapeutics at 1-888-400-SWTX (1-888-400-7989) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see full Prescribing Information for OGSIVEO for more information.

(Press release, SpringWorks Therapeutics, OCT 21, 2025, View Source [SID1234656824])

On October 21, 2025 Alligator Bioscience (Nasdaq Stockholm: ATORX), a clinical-stage biotechnology company developing tumor-directed immuno-oncology antibody drugs, reported that the United States Patent and Trademark Office (USPTO) has granted US Patent No. 12,448,465, covering the composition of matter of ATOR-4066, the company’s bispecific antibody targeting CD40 and CEACAM5. The patent will formally issue on 21 October 2025.

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The granted patent provides broad protection for ATOR-4066, including the bispecific antibody itself and its unique binding regions targeting CD40 and CEACAM5, and is expected to remain in force until 2043, excluding any potential patent term extensions.

ATOR-4066 is a novel bispecific antibody designed to activate CD40 on antigen-presenting cells while simultaneously targeting CEACAM5 on tumor cells. By bridging immune activation and tumor recognition, ATOR-4066 has demonstrated potent preclinical anti-tumor activity. The candidate is advancing toward clinical development as part of Alligator’s next-generation immuno-oncology pipeline. The program builds on Alligator’s extensive experience with mitazalimab, its lead CD40 agonist currently advancing toward pivotal development in pancreatic cancer.

"This patent grant marks an important milestone in strengthening the intellectual property protection around ATOR-4066," said Søren Bregenholt, CEO of Alligator Bioscience. "It underscores the innovation behind our bispecific antibody platform and reinforces the long-term value of our pipeline as we continue to advance novel immunotherapies toward the clinic."
Alligator has an additional continuation application pending in this patent family in the United States, further expanding the scope of protection around ATOR-4066.

(Press release, Alligator Bioscience, OCT 21, 2025, https://mfn.se/a/alligator-bioscience/alligator-bioscience-granted-us-patent-covering-ator-4066-bispecific-antibody [SID1234656861])

On October 21, 2025 Innovent Biologics (HKEX: 01801) reported a strategic global collaboration with Takeda (TSE:4502, NYSE:TAK) to advance next-generation IO and ADC cancer therapies, with the goal of developing potentially transformative cancer treatments to benefit patients worldwide.

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This partnership aims to leverage key synergies and accelerate the global development of several investigational medicines within Innovent’s IO+ADC pipeline, including: IBI363, a first-in-class PD-1/IL-2α-bias bispecific antibody fusion protein demonstrating robust anti-tumor activity and potential to be a foundational next-generation IO therapy that is currently in Phase 3 clinical stage; IBI343, a potentially best-in-class CLDN18.2 ADC currently in Phase 3 clinical stage; and IBI3001, a first-in-class EGFR/B7H3 bispecific ADC currently in Phase 1 clinical stage.

Dr. Hui Zhou, Chief R&D Officer for Oncology Pipeline at Innovent Biologics, stated,

"We believe that developing innovative IO and ADC will be a key direction for redefining cancer treatment worldwide. This landmark collaboration with Takeda brings together our three next-generation assets. With clear, aligned development plans, Innovent’s deep understanding of these assets, combined with Takeda’s extensive experience and strong development and commercialization capabilities, we are committed to delivering these promising medicines to patients worldwide as quickly as possible. This collaboration is also a crucial step in fulfilling Innovent’s strategic roadmap as we expand our global footprint, with the goal of becoming a leading global biopharmaceutical company."

"We are excited to partner with Innovent, an accomplished team with deep expertise in next-generation immuno-oncology and ADC biology," said Teresa Bitetti, President of the Global Oncology Business Unit at Takeda. "IBI363 and IBI343, two next-generation investigational medicines, have the potential to address critical treatment gaps for patients with a range of solid tumors. We are energized by the progress made by Innovent to date and look forward to collaborating to unlock the potential of these programs. Our global research and development expertise and commercialization capabilities will enable us to accelerate the delivery of these investigational medicines to patients. These two programs have the potential to be transformative for our oncology portfolio and significantly enhance Takeda’s growth potential post-2030."

IBI363 (PD-1/IL-2α-bias): Global Joint Development and Commercialization Collaboration

IBI363, developed by Innovent Biologics, is a potentially first-in-class PD-1/IL-2α-biased bispecific antibody fusion protein that simultaneously blocks the PD-1/PD-L1 pathway and activates the IL-2 pathway. Innovent has shown that IBI363, with an IL-2 receptor alpha focused approach, selectively expands tumor-specific CD8+ T cells that increase tumor cell killing efficiency without activating or expanding the toxicity related to peripheral T cells, which results in a better safety profile than what is seen with traditional IL-2s. Phase 1b/2 results presented at ASCO (Free ASCO Whitepaper) 2025 have demonstrated outstanding tumor responses and preliminary survival benefits of IBI363 across immunotherapy-resistant lung cancer, "cold tumors" such as acral and mucosal melanoma, and MSS colorectal cancer. IBI363 is now in registrational clinical development, including a global Phase 3 study in second line sqNSCLC that is expected to begin in the coming months; the China NMPA has granted Breakthrough Designation (BTD) and U.S. FDA has granted Fast Track Designation (FTD) for this indication.

According to the agreement, Innovent and Takeda will co-develop IBI363 globally, sharing development costs 40/60 (Innovent/Takeda). In the U.S., Innovent and Takeda will co-commercialize IBI363, sharing the U.S. profit or loss 40/60. Takeda will lead the co-development and co-commercialization efforts under joint governance and aligned development plan. In addition, Innovent will grant Takeda commercialization rights outside Greater China and the U.S. Takeda will have global manufacturing rights to supply IBI363 outside of Greater China, with such rights being co-exclusive with Innovent for commercial supply in the U.S. Takeda will pay Innovent potential development and sales milestones outside Greater China, and tiered royalties up to high-teens on net sales outside Greater China and the U.S.

This collaboration aims to explore and maximize IBI363’s potential as a new IO backbone therapy through aligned co-development plans. Building on its already robust clinical data of over 1,200 treated patients, IBI363 will be initially developed globally in non-small cell lung cancer ("NSCLC") and colorectal cancer ("CRC"), including in the first-line settings. Additionally, Takeda and Innovent plan to expand IBI363’s clinical development to additional indications.

IBI343 (CLDN18.2 ADC): Global License for Development and Commercialization

IBI343, developed by Innovent Biologics, is an innovative TOPO1 inhibitor ADC targeting CLDN18.2. Clinical data show a favorable safety profile and encouraging efficacy signals. It is currently being evaluated in a Phase 3 clinical trial in gastric/gastroesophageal cancers (G-HOPE-001) in China and Japan, and was granted Breakthrough Designation in China. IBI343 also completed a global Phase 1/2 trial in previously treated pancreatic ductal adenocarcinoma (PDAC) and has received Breakthrough Designation in China for this indication. It has also received Fast Track Designation from the U.S. FDA for the treatment of advanced unresectable or metastatic pancreatic ductal adenocarcinoma (PDAC) that has relapsed and/or is refractory to one prior line of therapy.

Innovent will grant Takeda exclusive global rights to develop, manufacture and commercialize IBI343 outside of Greater China. Takeda plans to advance the development of IBI343 and expand into first-line gastric and pancreatic cancer settings.

(Press release, Innovent Biologics, OCT 21, 2025, View Source [SID1234656878])

Takeda will make potential milestone payments, and tiered royalties on net sales up to high-teens for the license of IBI343.

IBI3001 (EGFR/B7H3 ADC): Option to Global License for Development and Commercialization

IBI3001, currently in a Phase 1 clinical trial, is a first-in-class bispecific ADC targeting B7-H3 and EGFR. It combines multiple anti-tumor mechanisms, including enhanced EGFR blockade, receptor-mediated internalization, and strong ADC-mediated cytotoxicity, with a high safety margin demonstrated in preclinical models.

Innovent will grant Takeda an exclusive option to license global development, manufacturing, and commercialization rights for IBI3001 outside of Greater China. If exercised, Takeda will pay Innovent an exercise fee, potential milestone payments, and tiered royalties on net sales up to mid-teens.

Financial Highlights: Total Deal Value up to $11.4Billion

Takeda will pay Innovent an upfront payment of US$1.2 billion, including a US$100 million equity investment in Innovent through new share issuance, at HK$112.56 per share, a 20% premium to the Innovent 30-trading-day weighted average share price.

Furthermore, Innovent is eligible for development and sales milestone payments for IBI363, IBI343, and IBI3001 (if option exercised) totaling up to approximately $10.2 billion, for a total deal value of up to $11.4 billion. Innovent will also receive potential royalty payments for each molecule outside Greater China, except with respect to IBI363 in the U.S., where the parties will share profits or losses.

Innovent will host conference calls and webcasts at 9:00 a.m. HKT (Chinese session) and 9:00 p.m. HKT (English session) on Wednesday, October 22, 2025.Details of the conference call dial-in and the webcast link will be provided on the company website at View Source A replay will also be available on the website shortly after the event.

Morgan Stanley Asia Limited serves as the exclusive financial advisor to Innovent Biologics in relation to this transaction.