On June 12, 2025 AIM ImmunoTech Inc. (NYSE American: AIM) (OTC Pink: AIMID) ("AIM" or the "Company") reported the United States Patent and Trademark Office has granted U.S. patent No. 12312376, titled "Therapeutic Double Stranded RNA and Methods for Producing the Same (Press release, AIM ImmunoTech, JUN 12, 2025, https://aimimmuno.com/aim-immunotech-granted-u-s-patent-covering-methods-of-manufacturing-therapeutic-dsrna-including-ampligen/ [SID1234653836])." The patent expires January 25, 2041.

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The new patent covers methods involving the manufacture of a range of therapeutic double-stranded RNA (dsRNA) products, of which Ampligen is included. Combined with AIM’s multiple compositions and methods patents involving Ampligen, this manufacturing patent, along with AIM’s other issued patents, further secures the Company’s control over the synthesis and use of the first-in-class drug, and provides patent protection for manufacturing until 2041.

The new patent significantly extends the potential development runway for the drug, securing AIM additional time to safely and confidently develop its drug program as it seeks U.S. Food and Drug Administration ("FDA") approval for Ampligen in any of a series of indications. Ampligen has demonstrated broad spectrum activity in clinical trials for globally important cancers, viral diseases and disorders of the immune system.

AIM’s overall intellectual property portfolio covers the manufacture and use of Ampligen. AIM’s patents include Ampligen in the treatment of cancer (U.S. patent 11,813,279, expires August 9, 2039), the Post-COVID condition of fatigue (Netherlands’ patent 2032813, expires August 21, 2042) and endometriosis (U.S. patent 12,102,649, expires October 22, 2040).

AIM CEO Thomas K. Equels commented, "This patent is a significant milestone for AIM as it represents the final step in a multi-year project to strengthen and secure our global patent portfolio surrounding Ampligen. AIM now has extended patent protection for the manufacture of Ampligen, the composition of Ampligen and the way in which Ampligen can be utilized as a therapeutic for a diverse collection of unmet medical needs, from deadly cancers to Post-COVID conditions to endometriosis."

Significantly, AIM’s intellectual property protection for Ampligen also includes multiple Orphan Drug Designations ("ODD") from the FDA and the European Medicines Agency ("EMA"). FDA designation grants seven years of market exclusivity after commercial approval and EMA designation grants 10 years of market exclusivity after such approval. AIM’s orphan drug designations include Metastatic Melanoma (US), Renal Cell Carcinoma (US), Pancreatic Adenocarcinoma (US and EU), Ebola Virus Disease (US and EU), Chronic Fatigue Syndrome/Myalgic Encephalomyelitis (US) and HIV (US).

On June 12, 2025 Debiopharm (www.debiopharm.com), a privately-owned Swiss biopharmaceutical company committed to establishing tomorrow’s standard of care to cure cancer and infectious diseases and Alkyon Therapeutics, Inc., a biotechnology company pioneering precision-targeted therapies, reported the signing of a co-research agreement to evaluate the feasibility of developing targeted radioligand therapies (RLTs) using Debiopharm’s proprietary AbYlink conjugation technology for Alkyon’s modular antibody platform directed against undisclosed tumor-associated antigens (Press release, Debiopharm, JUN 12, 2025, View Source [SID1234653868]).

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This collaboration marks a promising step toward a new generation of RLTs designed to deliver potent radiation targeted directly to cancer cells while minimizing damage to healthy tissues. By leveraging the unique strengths of both platforms, the goal is to create more effective, better-tolerated cancer treatments that improve patient quality of life and expand therapeutic options for difficult-to-treat tumors. AbYLink technology enables regio-selective lysine conjugation, ensuring the radioligand avoids the target binding site and facilitating streamlined, consistent manufacturing.

"We’re intrigued to explore the potential of applying AbYlink conjugation technology to the radio-oncology field," expressed Frederic Levy, Chief Scientific Officer, Debiopharm. "Leveraging our innovative conjugation platform with Alkyon’s engineered antibody scaffolds could open new possibilities for next-generation radiopharmaceuticals with enhanced precision and therapeutic impact."

"This collaboration reflects our shared commitment to expanding the potential of radioligand therapies," said Benjamin Titz, Co-Founder and CEO of Alkyon Therapeutics. "By applying AbYlink to our specialized antibody scaffolds and targeting strategies tailored to the unique architecture of solid tumors, we aim to unlock new therapeutic possibilities and advance care for patients with difficult-to-treat solid tumors."

About AbYlink

AbYlink is a versatile and rapid regio-selective chemical conjugation technology for use in preparing diagnostic or therapeutic conjugates. This one-step method results in stable conjugation at defined and invariable sites on the Fc domain of an antibody or the like, with no impact on antigen-binding regions. It enables a seamless and reproducible conjugation of payloads (e.g., a chelator for radiolabeling, a fluorescent dye or a drug) to antibodies or ADCs. The universal applicability of the technology has been demonstrated for various antibody isotypes and payloads.

On June 12, 2025 Aptose Biosciences Inc. ("Aptose" or the "Company") (TSX: APS; OTC: APTOF), a clinical-stage precision oncology company, reported data from its Phase 1/2 TUSCANY trial in newly diagnosed AML patients treated with tuspetinib (TUS) in combination with standard of care dosing venetoclax and azacitidine (TUS+VEN+AZA triplet) in an oral presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress (EHA 2025), being held June 12-15, 2025, in Milan, Italy (Press release, Aptose Biosciences, JUN 12, 2025, View Source [SID1234653837]).

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The TUS+VEN+AZA triplet is being developed as a mutation agnostic frontline therapy to treat large, mutationally diverse populations of newly diagnosed AML patients who are ineligible to receive induction chemotherapy. Dr. Gabriel Mannis, Associate Professor of Medicine, Stanford University School of Medicine, and an investigator in the TUSCANY study, reported safety and efficacy data from the first two dose cohorts at 40 mg of TUS or 80 mg of TUS in the TUS+VEN+AZA triplet. Dr. Mannis also noted three patients were rapidly enrolled on the third dose cohort of 120 mg TUS in the TUS+VEN+AZA triplet, and that no DLTs have been observed to date.

The oral presentation at EHA (Free EHA Whitepaper) included updated safety, complete remission, minimal residual disease (MRD) assessments, and longer duration of follow-up:
Title: TUSCANY Study of Safety and Efficacy of Tuspetinib Plus Standard of Care Venetoclax and Azacitidine in Study Participants with Newly Diagnosed AML Ineligible for Induction Chemotherapy
Presenter: Dr. Gabriel Mannis, Associate Professor of Medicine, Stanford University School of Medicine
Abstract #: S139

Key findings:

To date, ten newly diagnosed AML patients have received the TUS+VEN+AZA combination:
Four received the 40 mg dose of TUS, three received the 80 mg dose of TUS, and three received the 120 mg dose of TUS
At the initial dose of 40 mg TUS (n=4), with patients on longest duration of drug:
Three subjects achieved CRs and were MRD-negative, including
Patient with FLT3-ITD
Patient with FLT3-WT
Patient with TP53/CK
At the 80 mg TUS dose level (n=3):
All three patients (100%) already achieved composite complete remissions (CR and CRi)
A TP53-mutated/CK AML patient achieved an early CRi
Too early in treatment for final MRD assessment
At the 120 mg TUS dose level (n=3):
All three patients at the 120 mg TUS dose level remain on therapy
Too early in treatment for formal response and MRD assessments
Regardless of mutation status, TUS is active in newly diagnosed AML patients
MRD-negative responses achieved across diverse genetic populations, including adverseTP53 mutations and CK
Responses continue to evolve, and the triplet continues to be well tolerated with no DLTs
TUS can be administered safely with standard-of-care dosing of VEN/AZA
TUS PK properties not altered by VEN, AZA, antifungals or food
No prolonged myelosuppression in Cycle 1 in the absence of AML
No treatment-related deaths; all 10 subjects treated to date remain alive
No treatment related QTc prolongation, CPK elevations, differentiation syndrome or non-hematologic SAEs
"The TUSCANY triplet trial is well under way, and we are observing exciting activity with the addition of TUS to the VEN+AZA standard treatment," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer of Aptose. "The data presented today reveal complete responses across patients with diverse mutations, including TP53-mutated/CK AML and FLT3-wildtype AML patients. TUS appears to have tremendous opportunity in the largest markets and the most challenging of AML cases."

Abstracts are available on the EHA (Free EHA Whitepaper)2025 website here. The presentation is available on the Aptose website here.

TUSCANY: TUS+VEN+AZA Triplet Phase 1/2 Study

The tuspetinib-based TUS+VEN+AZA triplet therapy is being advanced in the TUSCANY Phase 1/2 clinical study with the goal of creating an improved frontline therapy for newly diagnosed AML patients that is active across diverse AML populations, durable, and well tolerated. Earlier APTIVATE trials of TUS as a single agent and in combination as TUS+VEN demonstrated favorable safety and broad activity in diverse relapsed or refractory (R/R) AML populations that went beyond the more prognostically favorable NPM1 and IDH mutant subgroups. Indeed, responses were also in R/R AML patients with highly adverse TP53 and RAS mutations, and those with mutated or unmutated (wildtype) FLT3 genes.

The TUSCANY Phase 1/2 study, being conducted at 10 leading U.S. clinical sites by elite clinical investigators, is designed to test various doses and schedules of TUS in combination with standard dosing of AZA and VEN for patients with AML who are ineligible to receive induction chemotherapy. A convenient, once daily oral agent, TUS, is being administered in 28-day cycles. Multiple U.S. sites are enrolling in the TUSCANY trial with anticipated enrollment of 18-24 patients by mid-late 2025. Data will be released as it becomes available.

On June 12, 2025 Blueprint Medicines Corporation (Nasdaq: BPMC) reported data presentations reflecting over a decade of collaboration with clinical experts and patient advocates to transform the treatment of systemic mastocytosis (SM) (Press release, Blueprint Medicines, JUN 12, 2025, View Source [SID1234653853]). Key results continue to position AYVAKIT/AYVAKYT (avapritinib) as the durable standard of care across indolent and advanced SM, and highlight the real-world burden of the disease, reinforcing the importance of treating with a therapy that addresses the root cause of SM. These data will be reported at the 2025 European Hematology Association (EHA) (Free EHA Whitepaper) (EHA2025) Hybrid Congress, being held June 12 to 15 in Milan, Italy, and the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025, being held June 13 to 16 in Glasgow, United Kingdom.

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"Our presentations feature large patient populations from the PIONEER, PATHFINDER and EXPLORER trials, with follow-up reaching up to five years in ISM and up to 6.5 years in advanced SM, reflecting both the favorable long-term benefits of AYVAKIT and the unprecedented datasets we have amassed over time," said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. "AYVAKIT has shown transformative clinical outcomes for patients across the spectrum of SM, including sustained disease control in ISM and prolonged survival in advanced SM. These compelling results have translated into real-world practice, with clinicians expanding their view of who is an appropriate candidate for disease-modifying therapy after positive AYVAKIT experiences, and treatment durations trending toward multiple years."

PIONEER Three-Year Data: Durable Clinical Benefits and Consistent Safety Profile with Long-Term AYVAKIT Use in ISM

As previously presented,1 AYVAKIT demonstrated robust improvements through 144 weeks in overall symptom and symptom domain measures (skin, gastrointestinal, neurocognitive) representative of real-world patient impacts. AYVAKIT showed a well-tolerated safety profile and a low discontinuation rate due to treatment-related adverse events (TRAEs; 3 percent) with a median of three years of exposure, and some patients out to five years on therapy. Common TRAEs included low-grade edemas, headache and nausea.
In newly reported data, AYVAKIT showed sustained clinical benefits across quality-of-life measures that reflect general health status and are broadly recognized by allergists/immunologists, validating previously presented results from the disease-specific, Mastocytosis Quality of Life (MC-QoL) questionnaire.
This data presentation follows the May 2025 online publication of PIONEER two-year efficacy and safety data in The Journal of Allergy and Clinical Immunology: In Practice.

PATHFINDER/EXPLORER Multi-Year Data: Long-Term Survival Benefits of AYVAKIT in Advanced SM

AYVAKIT showed prolonged overall survival (OS) in PATHFINDER and EXPLORER, when indirectly compared to real-world data for midostaurin from the German Registry on Disorders of Eosinophils and Mast Cells (GREM).
AYVAKIT led to meaningful survival benefits in patients across all prognostic categories (low, intermediate and high risk), per the Revised Mutation-Adjusted Risk Score (MARS-R) – a new OS risk assessment tool for advanced SM.
Conducted in collaboration with University Hospital Mannheim, the analyses validate the MARS-R tool’s ability to assess OS risks in advanced SM patients treated with AYVAKIT or midostaurin, using clinical and genetic parameters. The MARS-R was developed to inform physician care decisions based on individual patient needs.
PRISM Data: Substantial Disease Burden Across Broad Population of Patients with ISM

PRISM is one of the largest studies characterizing the impact of SM from both patient and clinical perspectives.
Across the spectrum of disease severity, patients with ISM experienced physical, social and emotional challenges that caused meaningful disruption to their daily lives.
Patients reported a broad constellation of disease-related impacts, including limitations to physical activities, work/college and relationships; problems with pain/discomfort and anxiety/depression; and adjustments in their daily lives to avoid certain foods, extended sun exposure and smells.
Data Presentations

EHA2025 Congress

Oral Presentation: The Revised Mutation-Adjusted Risk Score (MARS-R) for Predicting Overall Survival in Patients with Advanced Systemic Mastocytosis Treated with Midostaurin or Avapritinib (Abstract S216)
Poster Presentation: Blood-Based Proteomics for Deeper Insights Into Indolent Systemic Mastocytosis: The PIONEER Trial Experience (Abstract PS1838)
Poster Presentation: High Accuracy of Peripheral Blood Testing Using Machine Learning–Derived Predictive Models to Distinguish Advanced from Indolent Systemic Mastocytosis: Analysis of Avapritinib and Elenestinib Trial Data (Abstract PF1310)
Publication-Only Abstract: Phase 2/3 HARBOR Study of Elenestinib in ISM: A Trial-in-Progress Update of Novel Endpoints and Biomarkers Aimed at Evaluating Disease Modification (Abstract PB3108)
EAACI Congress 2025

Flash Talk Presentation: Favorable Benefit-Risk Profile of Avapritinib in Indolent Systemic Mastocytosis Is Maintained After 3 Years of Therapy: Longer-Term Analysis of the PIONEER Study (Abstract 000621)
Flash Talk Presentation: The Socio-Emotional Impact of Indolent Systemic Mastocytosis: Insights from the PRISM Survey (Abstract 000488)
Poster Presentation: The Phase 2/3 Study of Elenestinib, a Highly Potent and Selective Tyrosine Kinase Inhibitor, in Patients with Indolent Systemic Mastocytosis (Abstract 001121)
Data presentations are being made available in the "Science―Publications and Presentations" section of the company’s website at www.blueprintmedicines.com.

On June 12, 2025 Schrödinger, Inc. (Nasdaq: SDGR) reported encouraging initial clinical data from its ongoing Phase 1, open-label, dose-escalation study of SGR-1505 in patients with relapsed/refractory B-cell malignancies (Press release, Schrodinger, JUN 12, 2025, View Source [SID1234653869]). SGR-1505 was observed to be safe, well tolerated, and clinically active, with responses observed in multiple histologies, including in patients with chronic lymphocytic leukemia (CLL) and Waldenström macroglobulinemia (WM). These data are being presented in a poster presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress.

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"We are very encouraged by the initial results from our Phase 1 study in patients with relapsed/refractory B-cell malignancies. The data presented today, coupled with the differentiated preclinical and safety profiles observed in our previously completed study in healthy volunteers, further increases our conviction about the potential for SGR-1505 to be a best-in-class therapy," said Margaret Dugan, M.D., chief medical officer at Schrödinger. "Dose escalation is complete, and we look forward to discussing these results and our proposed recommended Phase 2 dose with the FDA later this year."

"Despite recent advances in the treatment of B-cell malignancies, resistance to currently available therapies eventually results in treatment failure and disease progression for many patients," said Stephen Spurgeon, M.D., Associate Professor of Medicine, Oregon Health and Science University, and an investigator for the clinical study. "We know that MALT1 plays a critical role in key signaling pathways that drive cancer cell survival and proliferation, making it a promising target for a broad range of B-cell malignancies. Although these data are from an early-phase study, they suggest SGR-1505 demonstrates on-target activity resulting in potential clinical benefit. I look forward to seeing additional data as the study progresses, including response data in patients with aggressive histologies."

"The positive data reported today represent a key milestone for Schrödinger and follow the clinical successes of programs advanced by collaboration partners and companies we have co-founded," said Karen Akinsanya, Ph.D., president, head of therapeutics R&D and chief strategy officer, partnerships at Schrödinger. "These data reinforce the power of Schrödinger’s platform to enable the rapid design of differentiated molecules and the impact that our computational approach can have on a drug discovery and development program."

Major Takeaways from the Study

As of the data cut-off date, May 13, 2025, 49 patients were enrolled and evaluable for safety, including 18 patients with CLL/SLL, nine with diffuse large B-cell lymphoma (DLBCL), six with Waldenström macroglobulinemia (WM), and five with marginal zone lymphoma (MZL).
Patients had a median of four (range two-nine) prior lines of therapy, with the most common being Bruton’s tyrosine kinase (BTK) inhibitors (55.1%), BCL-2 inhibitors (18.4%) and BTK+BCL-2 inhibitors (18.4%).
SGR-1505 was well-tolerated with no dose-limiting toxicities or deaths due to treatment-emergent adverse events (TEAEs). Forty three percent of patients (n=21) experienced ≥ 1 treatment-related adverse event (TRAE), with the most common (≥ 10%) being rash (12%) and fatigue (12%). Ten patients (20%) experienced treatment-emergent serious adverse events (SAEs); one was treatment-related. All blood bilirubin increased TEAEs were asymptomatic, reported in patients with UGT1A1 polymorphisms and none were Grade 4.
Inhibition of IL-2 is a pharmacodynamic biomarker for target engagement and an exploratory endpoint in the study. Preliminary data indicated that SGR-1505 inhibits T-cell derived IL-2 upon ex vivo stimulation achieving the PD target of ~90% inhibition in the majority of PD-evaluable participants treated at ≥ 150 mg QD and all Q12H doses at steady state.
Preliminary efficacy data indicated SGR-1505 was clinically active as a monotherapy in a number of relapsed/refractory B-cell malignancies. Of the 49 participants, 45 patients had at least one follow-up disease assessment or disease progression and were evaluable for preliminary efficacy. The overall response rate (ORR) across all dose levels was 22% (n = 10/45). Thirteen of 49 patients had been on treatment for ≥120 days.
Among patients with indolent disease, 3/17 CLL/SLL, 5/5 WM, and 1/5 MZL patients responded. The responses of the three CLL responders were independently reviewed and confirmed, and two had a partial response (PR) with lymphocytosis (PR-L). Two of three CLL patients with partial responses were double-exposed to BTK and BCL-2 inhibitors, and all WM patients were exposed to BTK inhibitors.
The study recently began enrolling patients with aggressive lymphomas into the 300 mg QD and 100 mg Q12H cohorts. A PR was reported in one of four ABC-DLBCL patients.
Study Design
The Phase 1 dose-escalation study (NCT05544019) assessed SGR-1505 as a monotherapy treatment in patients with relapsed/refractory B-cell malignancies. The primary endpoint is the incidence and severity of adverse events and dose-limiting toxicities. Secondary endpoints include pharmacokinetic and pharmacodynamic measurements as well as objective response rate, duration of response and disease control rate.

EHA Poster Presentation Details
The full abstract (#PS1569) can be found online at www.ehaweb.org.

Poster Title: A Phase 1 study of SGR-1505, an oral, potent, MALT1 inhibitor for relapsed/refractory (R/R) B-cell malignancies, including chronic lymphocytic leukemia/small lymphocytic leukemia (CLL/SLL)
Presentation Date and Time: Saturday, June 14, 2025, 6:30-7:30PM CST (12:30-1:30PM ET)
Location: Poster Session 2

About SGR-1505
SGR-1505 is an oral investigational MALT1 inhibitor being evaluated for the treatment of relapsed/refractory B-cell malignancies. MALT1 plays a central role in key signaling pathways that drive cancer cell survival and proliferation, making its location downstream of BTK in the NF-κB signaling pathway an attractive target for the development of novel therapeutics for a potentially broad range of B-cell malignancies. In preclinical studies, SGR-1505 was observed to be highly potent and selective, and has demonstrated anti-tumor activity in preclinical models both as a monotherapy and in combination with BTK and BCL-2 inhibitors. There is also emerging therapeutic rationale supporting MALT1 inhibition as a potential treatment for inflammatory and autoimmune disorders.

SGR-1505 was designed using Schrödinger’s computational platform at scale and was discovered approximately 10 months after the company started its MALT1 program. Schrödinger believes that SGR-1505 is currently the most advanced MALT1 inhibitor known to be in clinical development and has both first-in-class and best-in-class potential. A Phase 1 study in patients with relapsed/refractory B-cell malignancies is ongoing (NCT05544019).

Webcast and Conference Call Information
Schrödinger will host a conference call on Thursday, June 12, 2025, at 8:00 a.m. ET to review the clinical opportunity for SGR-1505 and review the Phase 1 data presented at EHA (Free EHA Whitepaper). The live webcast can be accessed under "Events & Presentations" in the investors section of Schrödinger’s website, View Source To participate in the live call, please register for the call here. It is recommended that participants register at least 15 minutes in advance of the call. The archived webcast will be available on Schrödinger’s website for approximately 90 days following the event.