On September 9, 2025 Tscan therapeutics presented its corporate presentation (Presentation, TScan Therapeutics, SEP 9, 2025, View Source [SID1234655880]).

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On September 9, 2025 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported five-year follow-up results on overall survival (OS) from the Phase 3 EMPOWER-Lung 3 trial, which evaluated Libtayo (cemiplimab) plus platinum-based chemotherapy versus chemotherapy alone as a first-line treatment for adults with locally advanced or metastatic non-small cell lung cancer (NSCLC) with no EGFR, ALK or ROS1 aberrations (Press release, Regeneron, SEP 9, 2025, View Source [SID1234655879]). The late-breaking data will be presented in a mini oral session at the IASLC 2025 World Conference on Lung Cancer (WCLC) hosted by the International Association for the Study of Lung Cancer.

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"After more than five years of follow-up, the EMPOWER-Lung 3 trial continues to demonstrate sustained survival – with an impressive overall survival probability of 19.4% at five years – when Libtayo is added to chemotherapy in patients with advanced non-small cell lung cancer," said Ana Baramidze, M.D., Ph.D., Head of Clinical Research Department at Todua Clinic, Tbilisi, Georgia. "Long-term results across tumor histologies were also reported, including a notable 22.3-month median overall survival for squamous NSCLC patients. Collectively, these data underscore Libtayo’s utility across a variety of patient types, both as a single agent and in combination with chemotherapy."

The five-year results for Libtayo plus chemotherapy presented at WCLC add to the breadth of long-term data for Libtayo in advanced NSCLC, including five-year outcomes from the EMPOWER-Lung 1 trial that were presented at WCLC 2024 confirming durable survival benefit as monotherapy.

At this year’s WCLC, five-year efficacy results, with a median follow-up of 60.9 months, found Libtayo plus chemotherapy remained superior to chemotherapy alone:

21.1-month median OS versus 12.9 months, representing a 34% reduction in the risk of death (hazard ratio [HR]: 0.66; 95% confidence interval [CI]: 0.53–0.83). The five-year probability of survival was 19.4% for the Libtayo combination versus 8.8% for chemotherapy.
8.2-month median progression-free survival (PFS) versus 5.5 months, representing a 42% reduction in the risk of disease progression (HR: 0.58; 95% CI: 0.47–0.72).
43.6% objective response rate (ORR) versus 22.1%. The ORR included a complete response rate of 6.4% versus 0%.      
16.4-month median duration of response (DoR) versus 7.3 months.
Also presented at WCLC were exploratory subgroup analyses that demonstrated survival benefits for patients treated with Libtayo plus chemotherapy compared to chemotherapy alone regardless of tumor histology or PD-L1 expression level. Specific efficacy results included a:

22.3-month median OS among patients with squamous histology (n=133) versus 13.8 months (n=67), representing a 32% reduction in risk of death (HR: 0.68; 95% CI: 0.49–0.94).
19.4-month median OS among patients with non-squamous histology (n=179) versus 12.4 months (n=87), representing a 38% reduction in risk of death (HR: 0.62, 95% CI: 0.46–0.82).
24.0-month median OS among patients with PD-L1 ≥1% (n=217) versus 12.1 months (n=110), representing a 46% reduction in risk of death (HR: 0.54; 95% CI: 0.41–0.70).
The safety profile at five years remained consistent with previously reported data. The median duration of exposure was 39 weeks to Libtayo plus chemotherapy and 21 weeks to chemotherapy alone. Adverse events (AEs) of any grade occurred in 96.5% of Libtayo plus chemotherapy patients (49% ≥Grade 3) and 95% of chemotherapy patients (33% ≥Grade 3). The most common AEs of any grade occurring in at least 10% of Libtayo plus chemotherapy patients included anemia (46%), alopecia (38%), nausea (25%), increase of alanine aminotransferase (18%), arthralgia (18%), decreased appetite (18%), hyperglycemia (18%), increase of aspartate transaminase (16%), neutropenia (16%), fatigue (15%), constipation (14%), thrombocytopenia (14%), dyspnea (14%), asthenia (13.5%), vomiting (13%), decreased weight (13%), increased blood creatinine (13%), insomnia (12%), hypoalbuminemia (11%), diarrhea (11%). Among Libtayo patients, treatment-related adverse events were ≥Grade 3 in 30% and led to permanent discontinuation in 4.5% and death in 1%, compared to 18%, 1% and 0.7% in the chemotherapy arm, respectively.

About the Phase 3 Trial
The randomized, multicenter Phase 3 trial, called EMPOWER-Lung 3, investigated a first-line combination treatment of Libtayo and platinum-doublet chemotherapy, compared to platinum-doublet chemotherapy alone. The trial enrolled 466 patients with locally advanced or metastatic NSCLC, as well as squamous or non-squamous histologies across all PD-L1 expression levels and with no ALK, EGFR and ROS1 aberrations.

Patients were randomized 2:1 to receive either Libtayo 350 mg (n=312) or placebo (n=154) administered intravenously every 3 weeks for 108 weeks, plus platinum-doublet chemotherapy administered every 3 weeks for 4 cycles. The primary endpoint was OS, and key secondary endpoints were PFS and ORR. The probability of survival and PFS were calculated according to Kaplan-Meier estimates.

Notably, patients in the trial had a variety of baseline characteristics commonly considered difficult-to-treat. Among those enrolled, 43% had tumors with squamous histology, 67% had tumors with <50% PD-L1 expression, 15% had inoperable locally advanced disease not eligible for definitive chemoradiation, and 7% had pretreated and clinically stable brain metastases. Additionally, 84% of patients had an ECOG 1 performance status. ECOG performance status assesses patient ability to conduct daily living activities and prognosis on a scale of increasing severity ranging from 0 (no symptoms) to 5 (death).

On September 9, 2025 NKGen Biotech, Inc. (OTC: NKGN) ("NKGen" or the "Company"), a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous and allogeneic natural killer ("NK") cell therapeutics, reported the successful completion of its acquisition of a majority equity stake in NKMax Co., Ltd. ("NKMax"), a Korean biotechnology company, out of bankruptcy (Press release, NKGEN Biotech, SEP 9, 2025, View Source [SID1234655878]). This acquisition marks a transformative step for NKGen, granting full control over key global manufacturing infrastructure, intellectual property ("IP"), and exclusive commercialization rights, while solidifying the Company’s position as an independent and vertically integrated leader in NK cell therapeutics.

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This transaction is a culmination of a multi-year journey that has seen NKGen overcome significant challenges, emerge stronger, and continue its unwavering commitment to advancing groundbreaking cell therapies. Originally founded as a subsidiary of NKMax in 2017, NKGen has long benefited from its parent’s financial backing to drive the development of its innovative NK cell therapy platform. In October 2023, NKGen made the pivotal decision to go public in the U.S., securing a $25 million backstop commitment from NKMax to fund clinical development of NKGen’s flagship Alzheimer’s treatment. However, NKMax ultimately provided only $10 million of the $25 million backstop initially committed, marking the start of financial challenges for NKGen.

There were several unforeseen and unfortunate circumstances which led to the suspension of trading of NKMax shares on the KOSDAQ Korean Stock Exchange, and NKMax’s subsequent collapse into bankruptcy in 2024. This created significant financial challenges and uncertainty for NKGen which negatively impacted its own stock, ability to raise funds and eventual delisting from Nasdaq. In response to these challenges, NKGen’s leadership acted swiftly, with the support of AlpineBrook Capital GP 1 Limited ("AlpineBrook"), which recognized the potential of the combined companies. AlpineBrook provided the majority of the $17 million funding to NKGen for the purchase of NKMax shares. The entirety of the funding for the NKMax shares was provided by AlpineBrook and NKGen CEO Dr. Paul Y. Song, with terms to be finalized and disclosed in due course. This strategic partnership and infusion of capital are a testament to AlpineBrook’s confidence in the long-term growth and success of NKGen, now poised to leverage its newly acquired assets to drive future commercialization.

"This is more than an acquisition, it’s a turning point in our company’s history," said Paul Y. Song, M.D., Chairman and CEO of NKGen. "The past couple of years have been incredibly challenging, but we had an unwavering belief in the science behind our therapy and its true potential to change lives. This belief drove each of us, at every level of the company, to make sacrifices, stay committed, and keep building even though our future was very uncertain. With the support of AlpineBrook, who recognized our dedication and scientific promise, we have been blessed with the chance to now fully integrate NKMax’s capabilities and expertise into NKGen while taking full control of all IP and global distribution rights. We believe removing the uncertainty of the parent company overhang has also begun to generate renewed interest from U.S. and international investors alike and will allow us to enter a new era of opportunity and long-term growth. We believe the acquisition of NKMax will also allow us to rapidly expand and forge partnerships throughout Asia and the Middle East."

Dr. Song continued, "I am incredibly optimistic about the future of our lead therapeutic candidate, troculeucel, which has already been granted fast track designation by the U.S. Federal Drug Administration. We are consistently demonstrating cessation of disease progression and/or demonstrable cognitive improvements in over 90% of all Alzheimer’s patients treated to date and we are eagerly awaiting the completion of enrollment and generation of clinical and biomarker data from our double blinded randomized Phase 2 trial for moderate stage Alzheimer’s disease. With past challenges behind us, we are more focused and energized than ever, now turning our attention to advancing our clinical trials both in the U.S. and globally, with the goal of delivering this groundbreaking therapy to the patients who need it the most."

Looking ahead, we believe NKGen is well-positioned to accelerate the clinical development of its lead Alzheimer’s therapy, troculeucel, and expand its pipeline of NK cell therapies. With full ownership of NKMax’s manufacturing infrastructure and IP, the company expects to be primed for global commercial success and further strategic partnerships. The Company anticipates significant investor interest, as it enters a new chapter focused on long-term value creation for shareholders and patients worldwide.

About Troculeucel

Troculeucel is a novel cell-based, patient specific, ex vivo expanded autologous NK cell immunotherapeutic drug candidate. NKGen is developing troculeucel for the treatment of neurodegenerative disorders and a broad range of cancers. Troculeucel is the International Nonproprietary Name ("INN") for SNK01 assigned by the World Health Organization ("WHO"). The WHO INN approval of troculeucel establishes a universally recognized nonproprietary drug name for SNK01 and marks a significant step on NKGen’s journey toward bringing this therapy to market.

On September 9, 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 updates to its active site status and recruitment for 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 9, 2025, View Source [SID1234655877]). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a global approval trial, including sites in the US, Europe and the Middle East.

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To advance this trial to recruiting the 45th subject in Q4 2025 for its first unblinding for early data insights into the trial’s efficacy and safety endpoints, the Company has the following expectations for September:

Global Expansion: Expanding recruiting beyond Ukraine to include Spain, Georgia, Poland, Romania, Italy, Lithuania, and the US;
Site Expansion: Adding 8 new active sites by the end of September, increasing the total to 20 sites recruiting;
Recruitment Goals: Treating, enrolling, or screening a total of 20 subjects by the end of September; and
Data Insights: Setting a solid footprint to recruit the 45th subject in Q4 2025, allowing for unblinded preliminary data assessment regarding efficacy and safety upon data lock.
Walter Klemp, Chairman and CEO of Moleculin, commented, "Our team is diligently expanding our active site footprint across the US, EU, and neighboring regions. We are confident in our target to recruit the 45th subject by Q4 2025 and achieve our first data unblinding shortly thereafter, especially since the consistent message we are receiving from investigators is that they are very eager to enroll patients in the MIRACLE trial. Moving from our end of Phase 2 meeting with the FDA to screening 13 subjects in just over a year exemplifies our operational efficiency. Of these 13, 10 have been dosed, further supporting our confidence in the speed of recruitment for MIRACLE."

Mr. Klemp continued, "We expect to increase to over 30 active sites by year-end, as we move toward a second data unblinding for the conclusion of Part A of the trial. That will only require an additional 30 to 45 subjects, so we expect to conclude Part A in the first half of 2026, before commencing Part B of the trial. This level of early visibility is, we believe, exceptionally rare for a Phase 2B/3 trial and highly advantageous for investors."

The MIRACLE study is a Phase 2B/3 clinical trial whereby data from the 2B (Part A) portion will be combined with the Phase 3 (Part B) 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 will 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 recruitment of the first 45 subjects in the second half of 2025 with unblinding shortly thereafter, 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.

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. Annamycin also benefits from composition of matter patent protection through 2040 with the potential to extend that protection as far as 2045. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the EMA.

On September 9, 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 updates to its active site status and recruitment for 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 9, 2025, View Source [SID1234655877]). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a global approval trial, including sites in the US, Europe and the Middle East.

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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

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To advance this trial to recruiting the 45th subject in Q4 2025 for its first unblinding for early data insights into the trial’s efficacy and safety endpoints, the Company has the following expectations for September:

Global Expansion: Expanding recruiting beyond Ukraine to include Spain, Georgia, Poland, Romania, Italy, Lithuania, and the US;
Site Expansion: Adding 8 new active sites by the end of September, increasing the total to 20 sites recruiting;
Recruitment Goals: Treating, enrolling, or screening a total of 20 subjects by the end of September; and
Data Insights: Setting a solid footprint to recruit the 45th subject in Q4 2025, allowing for unblinded preliminary data assessment regarding efficacy and safety upon data lock.
Walter Klemp, Chairman and CEO of Moleculin, commented, "Our team is diligently expanding our active site footprint across the US, EU, and neighboring regions. We are confident in our target to recruit the 45th subject by Q4 2025 and achieve our first data unblinding shortly thereafter, especially since the consistent message we are receiving from investigators is that they are very eager to enroll patients in the MIRACLE trial. Moving from our end of Phase 2 meeting with the FDA to screening 13 subjects in just over a year exemplifies our operational efficiency. Of these 13, 10 have been dosed, further supporting our confidence in the speed of recruitment for MIRACLE."

Mr. Klemp continued, "We expect to increase to over 30 active sites by year-end, as we move toward a second data unblinding for the conclusion of Part A of the trial. That will only require an additional 30 to 45 subjects, so we expect to conclude Part A in the first half of 2026, before commencing Part B of the trial. This level of early visibility is, we believe, exceptionally rare for a Phase 2B/3 trial and highly advantageous for investors."

The MIRACLE study is a Phase 2B/3 clinical trial whereby data from the 2B (Part A) portion will be combined with the Phase 3 (Part B) 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 will 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 recruitment of the first 45 subjects in the second half of 2025 with unblinding shortly thereafter, 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.

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. Annamycin also benefits from composition of matter patent protection through 2040 with the potential to extend that protection as far as 2045. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the EMA.