On September 8, 2025 Pasithea Therapeutics Corp. (NASDAQ: KTTA) ("Pasithea" or the "Company"), a clinical-stage biotechnology company developing PAS-004, a next-generation macrocyclic MEK inhibitor reported that the external Safety Review Committee recommended that the Company’s Phase 1/1b open label study to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of PAS-004, in adult participants with neurofibromatosis type 1 (NF1) should proceed to Cohort 2, 8mg tablet, without modification (Press release, Pasithea Therapeutics, SEP 8, 2025, View Source [SID1234655833]). This recommendation was based on the review of the safety data from three patients from Cohort 1 and the absence of any dose limiting toxicities (DLT’s).

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"We are seeing substantial enrollment demand and have already enrolled the first three Cohort 2 patients," stated Dr. Tiago Reis Marques, Chief Executive Officer of Pasithea.

About the Phase 1/1b Clinical Trial in Adult NF1 Patients

The primary objective of the Phase 1/1b study (NCT06961565) is to evaluate the safety and tolerability of PAS-004 when administered for one 28-day treatment cycle in adult NF1 participants with at least one and up to two additional target plexiform neurofibromas (PNs) that are symptomatic and inoperable, incompletely resected, or recurrent. Secondary objectives are (i) to identify the recommended Part B dose ("RPBD") or Maximum Tolerated Dose (MTD) of PAS-004, (ii) to characterize the PK and PD profile of PAS-004, (iii) to evaluate the preliminary efficacy of PAS-004 on target PN volume, (iv) to evaluate the preliminary efficacy of PAS-004 on the size, appearance, and associated symptoms of cutaneous neurofibromas (CNs), and (v) to evaluate the impact of PAS-004 on quality of life ("QOL") and any physical symptoms attributed to the target PN. Experimental objectives are (i) to evaluate the impact of PAS-004 on QOL and any physical symptoms attributed to CNs, (ii) to evaluate the impact of PAS-004 on pain and function attributed to PNs, and (iii) to investigate PAS-004 effects on CN tumor cellular and molecular biology.

The trial will be conducted in two parts. In Part A, following a screening period of up to 28 days, up to 24 eligible participants will be enrolled sequentially to receive one of four planned dose levels of PAS-004 tablets (4mg, 8mg, 12 mg, 18mg) in a modified 3+3 design. Part A will identify the recommended RPBD. During Part B, approximately 24 eligible participants will be enrolled in parallel to receive one of two planned dose levels of PAS-004 tablets. Participants will be dosed at the RPBD level and at a dose level below the RPBD for up to six continuous 28-day treatment cycles. Part B will identify the recommended phase 2 dose (RP2D).

The study is planned to be conducted at five clinical trial sites in Australia, South Korea, and the U.S.

On September 8, 2025 Oncolytics Biotech Inc. (Nasdaq: ONCY) ("Oncolytics" or the "Company"), a clinical-stage immunotherapy company developing pelareorep, reported clinical and translational data from three metastatic colorectal cancer ("mCRC") studies demonstrating consistent efficacy signals, immune activation, and survival outcomes that exceed historical benchmarks in multiple mCRC treatment settings (Press release, Oncolytics Biotech, SEP 8, 2025, View Source [SID1234655832]).

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In the REO 022 trial, pelareorep in combination with FOLFIRI and bevacizumab achieved the following results in platinum refractory 2L mCRC KRAS mutant patients:
•Median progression-free survival ("PFS"): 16.6 months vs. 5.7 months with standard 2L regimen1
•Median overall survival ("OS"): 27.0 months vs. 11.2 months with standard 2L regimen1

In the GOBLET study’s 3L mCRC Cohort 3, pelareorep combined with atezolizumab and TAS-102 met its predefined efficacy endpoint. The regimen achieved durable disease control and survival rates greater than historical benchmarks for 3L mCRC treated with TAS-102.

In the REO 022 trial and the REO 013 translational study, viral replication and immune activation were demonstrated in tumors from mCRC patients, including dendritic cell maturation and CD8+ T cell activation. These findings confirm pelareorep’s mechanism of action, including its ability to modify mCRC tumors to be immune responsive and amenable to checkpoint inhibition.

"These studies validate pelareorep’s mechanism of action and present a clear opportunity to accelerate the pursuit of a registration-enabled study in the underserved KRAS mutant subset of mCRC patients," said Jared Kelly, CEO of Oncolytics. "Given pelareorep’s activity in this difficult-to-treat cancer and other RAS-mutated gastrointestinal ("GI") tumors, including metastatic pancreatic and anal cancers, we believe pelareorep is positioned to become the premier platform immunotherapy in the GI space."

"The efficacy data for pelareorep in the hard-to-treat KRAS mutant population of mCRC are very encouraging," said Dr. Sanjay Goel, professor and attending physician at Rutgers University. "We plan to initiate an investigator-sponsored trial to further explore pelareorep’s promising potential in KRAS mutant mCRC, building on the robust immune activation demonstrated in REO 013 and the survival benefit seen in REO 022."

In addition to establishing an executable investigator-sponsored trial, the company plans to work with leading investigators and engage with regulators to define a clear path to registration in mCRC, including the design of a confirmatory study, leveraging the data in the KRAS mutant patient setting.

On September 8, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat cancers and viral infections, reported that it has enrolled the first two subjects, and treated one, in the European Union (EU) in its pivotal Phase 2B/3, multi-center, randomized, double-blind, placebo-controlled, adaptive design study of Annamycin in combination with cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as "AnnAraC") for the treatment of adult patients with acute myeloid leukemia (AML) who are refractory to or relapsed (R/R) after induction therapy (R/R AML) (Press release, Moleculin, SEP 8, 2025, View Source [SID1234655831]). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a global approval trial, including sites in the US, the EU, and other parts of Europe.

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"We now have active sites recruiting in the US, Spain, Ukraine, Georgia, and Romania with more sites expected to come online by the end of September. For our first site in Spain to have opened up with two subjects enrolled, we believe, indicates the unmet need in treating second line R/R AML," said Walter Klemp, Chairman and CEO of Moleculin. "All of this, importantly, supports our goal to recruit the first 45 subjects for Part A before the end of 2025 for which efficacy and safety will be unblinded."

The MIRACLE study is a Phase 2B/3 clinical trial whereby data from the 2B portion will be combined with the Phase 3 portion for purposes of measuring its primary efficacy endpoint. MIRACLE is subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, utilizes an adaptive design whereby the first 75 to 90 subjects will be randomized (1:1:1) in Part A of the trial to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, which Annamycin doses were specifically recommended by the FDA in the Company’s end of Phase 1B/2 meeting.

The protocol for the MIRACLE trial allows for the unblinding of preliminary primary efficacy data (Complete Remission or CR) and safety/tolerability of the three arms at 45 subjects, in addition to the conclusion of Part A (at 75 to 90 subjects). The first early unblinding is expected to yield 30 subjects treated with Annamycin (190mg/m2 and 230 mg/m2) in combination with HiDAC and 15 subjects treated with just HiDAC plus placebo. The Company expects to reach the first unblinding (45 subjects) in the second half of 2025, in addition to the second unblinding, which is expected in the first half of 2026. This accelerated estimated timeline is due in part to the positive response the Company received in meetings during December with potential investigators regarding recruitment for the trial.

As previously announced with regard to the EU, the clinical trial approval with EMA was granted under the condition that the Company present results of appropriate nonclinical GLP studies before initiating the Phase 3 portion (Part B) of the study. Results will be submitted as a substantial modification to the existing approved CTA.

For Part B of the trial, approximately 220 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin (randomized 1:1). The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative.

Patient dosing has commenced, and the initial data readout is on track for the second half of 2025. For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756. Additionally, the clinical trial in the EU is on euclinicaltrials.eu and the reference identifier there is 2024-518359-47-00.

Annamycin, also known by its non-proprietary name of naxtarubicin, currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the EMA.

On September 8, 2025 Integra Therapeutics, a global leader in cutting-edge gene writing tools to improve the efficacy, precision and safety of advanced therapies, reported it has closed a €10.7 million pre Series A round of investment (Press release, Integra Therapeutics, SEP 8, 2025, View Source [SID1234655830]). The funds come from new investors like the EIC Fund —the venture capital arm of the European Innovation Council (EIC) with the European Investment Bank (EIB) as the investor of record—, which invested €4 million, and CDTI Innvierte of the Spanish Ministry of Science, Innovation and University, which contributed €2.7 million, as well as from existing investors AdBio Partners, Columbus Venture Partners, Invivo Partners and Takeda Ventures, which continue to support the company’s vision.

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The funds will be used to incorporate new advances into the FiCAT gene writing platform, validate various next generation CAR-T therapies in the preclinical phase and expand the FiCAT cell engineering capabilities to facilitate technology transfer to the pharmaceutical industry. In May, the company announced new, highly promising preclinical data for its FiCAT platform at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper).

Moreover, Integra is developing its first gene therapy for a rare pediatric liver disease, funded by an EIC Accelerator grant.

"Thanks to the support of our investors, we will continue leading innovation in cell and gene therapies and are getting closer to transforming the treatment of complex diseases like cancer, autoimmune diseases and rare diseases," explains Avencia Sánchez-Mejías, PhD, CEO and Co-Founder of Integra Therapeutics.

"We are happy to announce EIC Fund’s investment in Integra Therapeutics. We empower them to accelerate the development of groundbreaking therapies that have the potential to transform lives and advance the field of gene therapy on a global scale," says Svetoslava Georgieva, Chair of the EIC Fund Board.

On September 8, 2025 ImmunityBio, Inc. (NASDAQ: IBRX), a leading immunotherapy company, reported new paradigm changing findings from the Phase 2 QUILT-3.055 study, demonstrating ANKTIVA (nogapendekin alfa inbakicept-pmln) reverses lymphopenia (low natural killer and T cells) in patients with checkpoint inhibitor-resistant advanced non-small cell lung cancer (NSCLC), with this reversal resulting in significant prolonged median overall survival (mOS) (Press release, ImmunityBio, SEP 8, 2025, View Source [SID1234655829]). The results were presented (P1.11.78) on Sunday, September 7, at the IASLC 2025 World Conference on Lung Cancer in Barcelona, Spain. A video presentation of the data is available on X.com @DrPatSoonShiong.

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Severe lymphopenia (loss of cancer-killing natural killer cells and T cells), is defined by the absolute lymphocyte count (ALC) of less than 1,000 lymphocyte cells/µL. This lowered ALC count is caused by the side effects resulting from current standards of treatment with radiotherapy, chemotherapy and immunotherapy, and is associated with reduced survival across all tumor types.1,2 Although reduced ALC is a recognized risk factor in a many cancers, it has largely been overlooked because no therapies have been approved to address lymphopenia. ANKTIVA is the first therapy under investigation specifically for this purpose.

"These results present the clinical evidence that a low lymphocyte count, as measured by ALC levels, is an actionable, accessible biomarker to identify and treat therapy-induced lymphopenia in cancer patients with ANKTIVA and prolong overall survival," said Dr. Patrick Soon-Shiong, Founder, Executive Chairman and Global Chief Scientific and Medical Officer of ImmunityBio. "ANKTIVA is the first FDA-approved immunotherapy to stimulate natural killer cells, CD4+ CD8+ T cells and memory T cells, the very cells that are depleted resulting in lymphopenia, and that play a key role in the immune system’s fight against cancer. It is my belief that treating lymphopenia in patients with low NK and T cell count, is as essential as the well-established treatment of anemia (low red blood cells count) or of neutropenia (low neutrophil count), which occur as a consequence of chemotherapy, independent of tumor types. By taking this approach, we hope to herald in the paradigm change of treating the immune system itself and thereby allow the immune system to cure the cancer, regardless of its anatomical location. The long-term survival demonstrated in patients with bladder cancer and now in lung cancer provide evidence of the potential validity of this hypothesis, with ANKTVIA as the foundation of the BioShield platform."

Specific findings from the Phase 2 QUILT-3.055 study include:

Participants with a baseline ALC≥1,200 cells/µL and a group mean ALC≥1500 cells/µL at each on-treatment time point (N=44) had greater prolonged OS (21.1 months, 95% CI: 13.9. 42.1)
mOS for study participants with NSCLC, including patients in second line (51%) and third line-plus treatment (49%), was 14.3 months (95% CI 11.7, 17.4), with 23 of 86 participants alive at data cutoff (December 2024)
Among patients treated with ANKTIVA, 80% (69 of 86) exceeded an ALC of 1,200 cells/µL, with mOS of 15.8 months (95% CI 12.6, 21.9) compared to participants who failed to achieve ALC>1,200 cells/µL with mOS of 11.5 months (95% CI 4.2, 13.3); p=0.0057
60% of participants (25/42) treated with ANKTIVA reversed their lymphopenia during at least one on-treatment timepoint.
The randomized Phase 3 ResQ201A trial (NCT06745908) is evaluating ANKTIVA + tislelizumab, a checkpoint inhibitor, versus docetaxel alone in second-line NSCLC patients who have progressed after all standard therapies, including checkpoint inhibitors. Patients interested in enrolling can inquire at org.
The Phase 2 QUILT-3.055 study (NCT03228667) enrolled participants with advanced NSCLC who had developed resistance to checkpoint inhibitor immunotherapy (CPI). Participants continued treatment with the same CPI on which they had progressed, combined with ANKTIVA, to test the hypothesis that AKNTIVA could improve overall survival by reversing lymphopenia and maintaining median ALC≥1,200 cells/µL. The primary endpoint was the change in ALC, including absolute cell count and percentage change from baseline, with ANKTIVA plus CPI during the study and its relationship to mOS. Statistical differences between OS among participants who failed to reverse lymphopenia (ALC<1,200 cells/µL) during treatment vs. those who had a baseline ALC≥1200 cells/µL and maintained a group mean ALC≥1,500 cells/µL while on treatment were compared. The study also assessed the proportion of participants with lymphopenia reversal (baseline ALC<1,200 cells/µL and at least one on-treatment ALC≥1,200 cells/µL).

ANKTIVA is currently approved by the U.S. Food and Drug Administration with Bacillus Calmette-Guérin (BCG) for the treatment of patients with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors.

About ANKTIVA (nogapendekin alfa inbakicept-pmln)

The cytokine interleukin-15 (IL-15) plays a crucial role in the immune system by affecting the development, maintenance, and function of key immune cells—NK and CD8+ killer T cells—that are involved in killing cancer cells. By activating NK cells, ANKTIVA overcomes the tumor escape phase of clones resistant to T cells and restores memory T cell activity with resultant prolonged duration of complete response. A key component in the company’s BioShield platform, ANKTIVA is a first-in-class IL-15 agonist IgG1 fusion complex, consisting of an IL-15 mutant (IL-15N72D) fused with an IL-15 receptor alpha, which binds with high affinity to IL-15 receptors on NK, CD4+, and CD8+ T cells. This fusion complex of ANKTIVA mimics the natural biological properties of the membrane-bound IL-15 receptor alpha, delivering IL-15 by dendritic cells and driving the activation and proliferation of NK cells with the generation of memory killer T cells that have retained immune memory against these tumor clones.

IMPORTANT SAFETY INFORMATION

INDICATION AND USAGE: ANKTIVA is an interleukin-15 (IL-15) receptor agonist indicated with Bacillus Calmette-Guérin (BCG) for the treatment of adult patients with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

WARNINGS AND PRECAUTIONS: Risk of Metastatic Bladder Cancer with Delayed Cystectomy. Delaying cystectomy can lead to the development of muscle-invasive or metastatic bladder cancer, which can be lethal. If patients with CIS do not have a complete response to treatment after a second induction course of ANKTIVA with BCG, reconsider cystectomy.

DOSAGE AND ADMINISTRATION: For Intravesical Use Only. Do not administer by subcutaneous or intravenous routes.

Please see the complete Indication and Important Safety Information and Prescribing Information for ANKTIVA at Anktiva.com.