On August 18, 2025 Aptose Biosciences Inc. ("Aptose" or the "Company") (OTC: APTOF, TSX: APS), a clinical-stage precision oncology company developing the tuspetinib (TUS)-based triple drug frontline therapy to treat patients with newly diagnosed AML, reported a data update from the Phase 1/2 TUSCANY trial in newly diagnosed AML (Press release, Aptose Biosciences, AUG 18, 2025, View Source [SID1234655342]). The trial was initiated in December 2024, and the growing body of positive data includes the recently completed third cohort of 120 mg TUS in the TUS+VEN+AZA triplet therapy. Data to date from ten (10) patients across all three cohorts, 40 mg, 80 mg or 120 mg TUS dose in TUS+VEN+AZA, support the use of TUS with standard of care treatment across all AML populations, including those carrying mutations that are the most difficult to treat and those with mutated and unmutated (wildtype) FLT3 genes.

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 TUS+VEN+AZA triplet is being developed as a safe and well-tolerated mutation agnostic frontline therapy to treat large, mutationally diverse populations of newly diagnosed AML patients who are ineligible to receive induction chemotherapy. At the 120 mg TUS dose level in combination with VEN+AZA, as with the prior reported 40 mg and 80 mg TUS dose cohorts, no significant safety concerns or dose limiting toxicities (DLTs) have been observed in the TUSCANY trial, including no prolonged myelosuppression in Cycle 1 of subjects in remission, no reports of drug-related QTc prolongation or differentiation syndrome (DS), and no treatment-related deaths. Nine out of ten dosed patients remain on study across all dose cohorts and enrollment is being advanced to the 160 mg TUS dose level following the Cohort Safety Review Committee (CSRC) meeting.

"We already have data from three different TUS dose levels in the TUSCANY trial, and the data continue to strengthen at higher doses of TUS and over time. We are building a strong case for TUS+VEN+AZA as a triplet frontline therapy of choice to address a broad AML population, including subgroups with the most adverse of mutations," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer of Aptose.

Data highlights:

Comparison of CR/CRh1 Response rates2-5:

VEN+AZA2 TUS+VEN+AZA
All subjects 65% 90% (9/10)
NPM1-mutant 67% 100% (2/2)
FLT3-ITD 61% 100% (2/2)
TP53-mutant 52% 100% (2/2)

Comparison of MRD-negativity6 rates among All Subjects and among CR/CRh Responders3:

VEN+AZA2,3 TUS+VEN+AZA
Among All Subjects 23.4% 70% (7/10)
Among CR/CRh Responders 40.9% 78% (7/9)

Comparison of MRD-negativity rates among more difficult-to-treat Patient Subpopulations defined as Lower Benefit (TP53-mutated) and Intermediate Benefit (FLT3-ITD or RAS-mutated) relative to VEN+AZA5:

VEN+AZA3,5 TUS+VEN+AZA
Intermediate Benefit 27.9% 100% (3/3)
Lower Benefit 14.5% 100% (2/2)

TUS+VEN+AZA – CR/CRh and MRD-negativity rates among Subjects with Adverse Mutations:

TP53, FLT3-ITD, RAS mutations: Achieved CR/CRh and MRD-negativity
100% (5/5)

"As illustrated in the data highlights, the addition of TUS to VEN+AZA appears to boost response rates and MRD-negativity while maintaining favorable safety and tolerability," said Rafael Bejar, M.D., Ph.D., Chief Medical Officer of Aptose, "and the 100% CR/CRh and 100% MRD-negativity rates among the five biallelic TP53-mutant, FLT3-ITD, and RAS-mutant AML cases are exciting to see, as this can correlate with longer overall survival. We have observed a trend towards achieving CRs more quickly at the higher dose levels, so we are keen to see the activity as we advance into the 160 mg TUS dose cohort."

Key messages:

Addition of TUS to VEN+AZA demonstrates excellent CR/CRh rates
100% CR/CRh among all subjects treated at 80 mg and 120 mg TUS dose levels
Appear to be achieving CR earlier with 120 mg TUS than with 40 mg or 80 mg
Addition of TUS to VEN+AZA demonstrates excellent MRD-negativity rates
MRD-negativity in 7 of 9 (78%) already achieved in patients who responded to therapy
Expect patient survival to be extended with continued long-term treatment
Excellent safety and well tolerated with no dose-limiting toxicities (No DLT) at completed dose levels
Broad-spectrum activity including patients with adverse TP53, RAS and FLT3-ITD mutations
No loss of MRD-negativity observed to date, including in one patient with over 7 months of follow up
MRD-negativity and remissions continue to mature over time on therapy
No relapses reported to date and no treatment related deaths
The only non-responder was a patient at the initial TUS dose level (40 mg) that did not achieve TUS exposures previously associated with response
Additional data are included in the new Aptose corporate presentation 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 trial 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.

The TUSCANY triplet 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 late 2025. Data will be released as it becomes available.

More information on the TUSCANY Phase 1/2 study can be found on www.clinicaltrials.gov (here: View Source).

On August 18, 2025 Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, reported that the second patient in the fourth dose cohort has been treated in the ongoing Phase 1 clinical trial evaluating the Company’s novel chimeric antigen receptor-T cell (CAR-T) therapy for recurrent ovarian cancer (Press release, Anixa Biosciences, AUG 18, 2025, View Source [SID1234655341]). The study is being conducted through a research partnership with Moffitt Cancer Center ("Moffitt").

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!

Patients in the fourth cohort are receiving a dose of three million CAR-positive cells per kilogram of body weight—representing a 30-fold increase over the initial dose level. To date, no dose-limiting toxicities (DLTs) have been observed in the fourth cohort.

Anixa’s proprietary CAR-T program targets the follicle-stimulating hormone receptor (FSHR), which preclinical research indicates is selectively expressed on ovarian cells, tumor vasculature, and certain cancer cells—but not in healthy tissue. The ongoing first-in-human clinical trial (NCT05316129) is enrolling adult women with recurrent ovarian cancer who have progressed after at least two prior lines of therapy. The Phase 1 study is designed to evaluate safety, determine the maximum tolerated dose, and monitor initial signals of clinical activity.

"Our therapy continues to demonstrate a favorable safety profile, even at significantly higher cell doses," stated Dr. Amit Kumar, Chairman and CEO of Anixa Biosciences. "While this study is primarily designed to assess safety, we remain encouraged by early indications of potential efficacy as the trial progresses."

Anixa’s CAR-T technology was invented by Jose R. Conejo-Garcia, M.D., Ph.D., Professor of Immunology in the Department of Integrative Immunobiology at the Duke University School of Medicine. The ongoing clinical trial is being conducted at Moffitt under the direction of Dr. Robert Wenham, Chair of the Gynecologic Oncology Program. Anixa holds an exclusive worldwide license to the FSHR-targeting CAR-T technology from The Wistar Institute.

On August 18, 2025 Anocca AB (‘Anocca’ or the ‘Company’), a leading clinical-stage T-cell immunotherapy company, reported the company has successfully raised SEK ~440 million (USD ~46 million) in financing (Press release, Anocca, AUG 18, 2025, View Source [SID1234655340]). The additional capital will be used to drive the continued progress of VIDAR-1, Anocca’s gene-edited TCR-T cell therapies targeting mutant KRAS in pancreatic cancer, through early-stage clinical development, as well as progress Anocca’s preclinical pipeline. The financing was led by Mellby Gård with strong support from AMF, Ramsbury and existing shareholders, alongside new investors.

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!

Recruitment is now open for Phase I of the multi-centre trial of VIDAR-1, which is being conducted at leading university hospitals across Sweden, Denmark, Germany and The Netherlands.

Reagan Jarvis, co-founder and Chief Executive Officer of Anocca, said: "We thank our investors for their strong and continued support as we advance our first TCR-T cell therapy products into the clinic. The team has built a unique discovery platform and in-house manufacturing capability, and we are now excited to see the first products reaching patients with high unmet need."

Johan Andersson, Chairman of Mellby Gård, commented: "We have been invested in Anocca throughout its preclinical development and are pleased to see the Company transition into a clinical stage biotech. We look forward to seeing Anocca help patients that have limited treatment options today with their innovative approach to T-cell therapies".

SEB Corporate Finance acted as financial advisor to Anocca on the finalisation of the transaction, and Mannheimer Swartling and HWF Advokater have acted as legal advisors.

On August 18, 2025 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a commercial biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, reported Health Canada has issued a Notice of Compliance with Conditions (NOC/c) for Amtagvi (lifileucel), a tumor-derived autologous T cell immunotherapy (Press release, Iovance Biotherapeutics, AUG 18, 2025, View Source [SID1234655338]). Amtagvi is indicated for the treatment of adult patients with unresectable or metastatic melanoma that has progressed on or after at least one prior systemic therapy including a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitor, and who have no satisfactory alternative treatment options.

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!

"This approval in Canada is our first marketing authorization outside the U.S. and marks a significant step forward for Iovance as we prepare to introduce Amtagvi in countries with a high prevalence of advanced melanoma and address substantial unmet needs in solid tumor cancers," said Frederick Vogt, Ph.D., J.D., Interim Chief Executive Officer and President of Iovance. "We expect to authorize our first Canadian treatment center within the next few months, and we continue to advance our ex-U.S. strategy for Amtagvi in additional markets."

Market authorization in Canada under the NOC/c guidance was granted based on safety and efficacy results from the global, multicenter C-144-01 trial investigating Amtagvi in patients with advanced melanoma previously treated with anti-PD-1 therapy and targeted therapy, where applicable. The market authorization is conditional, pending the results of trials to confirm its clinical benefit.

About the C-144-01 Clinical Trial
C-144-01 is a global, multicenter Phase 2 study in which patients received treatment with lifileucel monotherapy. The study enrolled patients with metastatic melanoma who were previously treated with at least one systemic therapy, including a PD-1 blocking antibody, and if BRAF V600 mutation‑positive, a BRAF inhibitor or BRAF inhibitor with MEK inhibitor. Efficacy was established on the basis of objective response rate (ORR), and duration of response (DOR) by Independent Review Committee (IRC) per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1. The detailed results of C-144-01 were published in the Journal for ImmunoTherapy of Cancer in 2022. A five-year analysis of C-144-01 was published in the Journal of Clinical Oncology in 2025.

Iovance is investigating Amtagvi in frontline advanced melanoma in the Phase 3 trial, TILVANCE-301 (NCT05727904), as well as in additional solid tumor types.

On August 15, 2025 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the advancement of innovative precision medicines to improve the lives of patients, reported that the US Food and Drug Administration (FDA) has approved PAPZIMEOS (zopapogene imadenovec-drba) for the treatment of adults with recurrent respiratory papillomatosis (RRP). PAPZIMEOS is the first and only FDA-approved therapy for the treatment of adults with RRP. Precigen completed submission of the rolling Biologics License Application (BLA) in December 2024 under an accelerated approval pathway; however, the FDA has granted PAPZIMEOS full approval, which does not require a confirmatory clinical trial. PAPZIMEOS is a non-replicating adenoviral vector-based immunotherapy designed to express a fusion antigen comprising selected regions of human papillomavirus (HPV) types 6 and 11 proteins—the root cause of RRP. PAPZIMEOS is delivered via four subcutaneous injections over a 12-week interval.

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!

RRP is a rare, debilitating, and potentially life-threatening disease of the upper and lower respiratory tract caused by chronic HPV 6 or HPV 11 infection. RRP can lead to severe voice disturbance, a compromised airway, and recurrent post-obstructive pneumonias. Management of RRP has primarily consisted of repeated surgeries, which do not address the root cause of the disease and can be associated with significant morbidity as well as significant patient and health system burden.

"For more than a century, since RRP was first recognized as a distinct disease, patients have had to rely on repeated surgeries to manage this relentless condition. Today marks a historic turning point. With the landmark FDA approval of PAPZIMEOS and broad label, all adult RRP patients are now eligible for access to the first and only approved therapy that targets the root cause of the disease," said Helen Sabzevari, PhD, President and CEO of Precigen. "This milestone affirms the power of our AdenoVerse platform and the exceptional capabilities of our team to rapidly advance a wholly novel therapy from discovery to approval considerably faster than industry benchmarks. We are profoundly grateful to the NIH clinicians, the FDA, and—most importantly—the patients and families who made this breakthrough possible. We look forward to swiftly delivering PAPZIMEOS to the RRP community and ushering in a new era of treatment that targets the underlying cause of the disease rather than just managing its symptoms."

"This long-awaited FDA approval represents a momentous milestone for the RRP community," said Kim McClellan, President of the Recurrent Respiratory Papillomatosis Foundation. "For the first time, adult patients with RRP have access to an FDA-approved therapy that offers the potential to reduce—or even eliminate—endless repeated surgeries. This breakthrough brings long-overdue hope to patients and families who have endured so much. We are deeply grateful to the teams at Precigen and the NIH, and above all, to the patients and caregivers whose courage, advocacy, and perseverance have made this historic moment possible."

The approval is supported by data from the open-label, single-arm, pivotal study in adult patients with RRP:

The pivotal study successfully met its primary safety and pre-specified primary efficacy endpoints.
51% (18 out of 35) of study patients achieved Complete Response, requiring no surgeries in the 12 months after treatment with PAPZIMEOS. These Complete Responses remained durable for over 12 months. Of the 18 patients with a Complete Response in the ongoing study, 15 patients evaluated at 24 months demonstrated continued Complete Response.
PAPZIMEOS was well-tolerated with no dose-limiting toxicities and no treatment-related adverse events greater than Grade 2.
PAPZIMEOS induced HPV 6/11-specific T cell responses in RRP study patients with a significantly greater expansion of peripheral HPV-specific T cells in responders compared with non-responders.
The pivotal study was led by lead investigators, Clint T. Allen, MD, and Scott M. Norberg, DO, at the National Institutes of Health. Pivotal data were presented at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting and published in The Lancet Respiratory Medicine.

Precigen will begin promoting PAPZIMEOS immediately and is committed to helping patients with RRP access the therapy. Precigen has established Papzimeos SUPPORT, a comprehensive patient support program offering personalized services, including insurance navigation, financial assistance, and ongoing access support, which can be accessed by calling 866-827-8180. Healthcare professionals interested in learning more about PAPZIMEOS or accessing provider support services are encouraged to visit www.PAPZIMEOS.com.

Conference Call
The Company will host a conference call on Monday, August 18 at 8:00 AM ET to provide additional details regarding the approval, including key aspects of the label and commercialization. Event details can be found on Precigen’s website in the Events & Presentations section at investors.precigen.com/events-presentations.

About RRP
RRP is a rare, debilitating, and potentially life-threatening disease of the upper and lower respiratory tract caused by chronic HPV 6 or HPV 11 infection. RRP can lead to severe voice disturbance, compromised airway, and recurrent post-obstructive pneumonias. Although rare, RRP has the potential for transformation to malignant cancer and can be fatal. Management of RRP has primarily consisted of repeated surgeries, which do not address the underlying cause of the disease and can be associated with significant morbidity as well as significant patient and health system burden. As the number of lifetime surgeries increases, the risk for irreversible iatrogenic laryngeal injury increases with each surgery, and patients may undergo hundreds of these surgeries over their lifetimes. RRP can impact patients’ work and social lives, financial stability, and mental health. Patients with RRP can experience substantial impacts to daily living with decreased quality of life and high health care utilization. Based on an internal analysis of claims data and electronic health records, there are approximately 27,000 adult RRP patients in the US.

About PAPZIMEOS (zopapogene imadenovec-drba), for subcutaneous injection only
PAPZIMEOS is the first and only FDA-approved therapy for the treatment of adults with RRP and the first and only approved therapy to address the root cause of RRP. PAPZIMEOS is a non-replicating adenoviral vector-based immunotherapy designed to express a fusion antigen comprising selected regions of human papillomavirus (HPV) types 6 and 11 proteins. PAPZIMEOS is designed to generate an immune response directed against HPV 6 and HPV 11 proteins in patients with RRP. Discovered and designed in Precigen’s labs using Precigen’s proprietary AdenoVerse therapeutic platform, PAPZIMEOS represents a new therapeutic paradigm for RRP.

Indication and Important Safety Information

What is PAPZIMEOS?
PAPZIMEOS is a type of immunotherapy used to treat a condition called recurrent respiratory papillomatosis (RRP) in adults.

What is the most important information I should know about PAPZIMEOS?
Some people may have a reaction to the shot. Signs and symptoms may include redness, pain, swelling, itching, or warmth where the shot was given. After your first treatment, your healthcare provider will watch you for at least 30 minutes to make sure you’re feeling okay.

Please contact your doctor immediately if you develop an infection, the reaction to your shot worsens, or you experience any of the below symptoms, which may indicate a systemic allergic reaction:

Difficulty breathing
Widespread rash
Facial swelling
Thrombotic events (blood clots that block your blood vessels) may occur after your PAPZIMEOS shot. Please notify your doctor immediately if you have the following symptoms:

Shortness of breath
Chest pain
Leg swelling
Persistent abdominal pain
Severe or persistent headaches
Blurred vision
What should I know before taking PAPZIMEOS?
Before taking PAPZIMEOS, tell your healthcare provider about all of your medical conditions, including:

If you are pregnant or plan to become pregnant because it is not known if PAPZIMEOS will harm the unborn baby.
If you are breastfeeding or plan to breastfeed. It is unknown if PAPZIMEOS is present in breast milk, or how it affects the breastfeeding child or milk production. Talk to your healthcare provider about the best way to feed your baby during treatment with PAPZIMEOS.
What are the most common side effects of PAPZIMEOS?
The most common side effects include:

Pain, redness, or swelling where the shot was given
Feeling tired
Chills
Fever
Muscle aches
Nausea (feeling sick)
Headache
Increased heart rate
Diarrhea
Vomiting
Sweating a lot
These are not all of the possible side effects of PAPZIMEOS. Call your healthcare provider for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. You may also report side effects to Precigen, Inc. at 1-855-PGE-NRRP (1-855-743-6777).

(Press release, Precigen, AUG 15, 2025, View Source [SID1234662311])