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Kestrel Therapeutics Receives FDA Fast Track Designation for KST-6051, a Potential Best-in-Class Pan-KRAS Inhibitor, in the treatment of KRAS-Mutant Advanced Solid Tumors

On September 15, 2026 Kestrel Therapeutics Inc. ("Kestrel" or the "Company"), a clinical-stage biotechnology company developing next-generation small-molecule inhibitors targeting mutant KRAS, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to KST-6051, a potential best-in-class, oral pan-KRAS inhibitor, for the treatment of patients with advanced or metastatic solid tumors harboring KRAS mutations.

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Fast Track is a designation created to facilitate the development and expedite the review of drugs intended to treat serious conditions and address an unmet medical need. Companies that receive Fast Track designation benefit from more frequent communication with the FDA throughout development, and may be eligible for Accelerated Approval, Priority Review, and rolling review of a New Drug Application (NDA), provided relevant criteria are met.

"FDA Fast Track designation for KST-6051 is an important regulatory milestone for Kestrel, based on the strength of our preclinical data package. This designation will allow us to work more closely with the FDA as we advance KST-6051 through our ongoing Phase 1 study, with the goal of bringing a much-needed treatment option to patients with KRAS-mutant tumors as efficiently as possible," said Frank Haluska, MD, PhD, President and Chief Executive Officer of Kestrel Therapeutics.

KST-6051 is being evaluated in an ongoing first-in-human (FIH), dose-escalation Phase 1 study, denoted FALCON (NCT07458347) in patients with advanced or metastatic KRAS-mutant solid tumors.

About Fast Track Designation

The FDA’s Fast Track program is designed to facilitate development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to patients earlier. Fast Track addresses a broad range of serious conditions, and a drug that receives Fast Track designation is eligible for more frequent meetings and written communication with the FDA, as well as the possibility of Priority Review and rolling submission of a marketing application.

About KST-6051

KST-6051 is a potential best-in-class, oral pan-KRAS inhibitor, developed for the treatment of KRAS-driven cancers. KST-6051 is a potent and selective inhibitor of KRAS with activity against KRAS in both its active (GTP-bound) and inactive (GDP-bound) states. Preclinical data demonstrate robust on-target pathway modulation, anti-proliferative activity, and efficacy at well-tolerated doses in multiple human KRAS mutant tumor models. The Company is initiating a Phase 1 study in patients with KRAS-mutant advanced/metastatic solid tumors. Its clinical development will ultimately address pancreatic ductal adenocarcinoma (PDAC), colorectal cancer (CRC), non-small cell lung cancer (NSCLC), and other KRAS-driven malignancies.

(Press release, Kestrel Therapeutics, SEP 15, 2026, View Source [SID1234670885])

Ernexa Therapeutics Reports Positive Disease Modifying ERNA-201 Preclinical Data, Reinforcing iMSC Platform as Company Accelerates Oncology Strategy and ERNA-101 Toward First-in-Human Dosing

On September 15, 2026 Ernexa Therapeutics (Nasdaq: ERNA), an industry innovator developing novel cell therapies for the treatment of advanced cancers, reported positive preliminary preclinical results for ERNA-201, its synthetic, allogeneic induced mesenchymal stem cell (iMSC) therapy engineered to express anti-inflammatory factors, while outlining a sharpened oncology strategy designed to accelerate the Company’s transition into the clinic and expand the long-term potential of its proprietary synthetic iMSC platform.

In preliminary studies using the collagen-induced arthritis (CIA) model, an industry-standard preclinical model for rheumatoid arthritis and inflammatory joint disease, several ERNA-201 lead candidates demonstrated reductions in disease severity relative to untreated controls, including improvements in clinical arthritis scores consistent with the therapy’s intended anti-inflammatory mechanism of action.

Ernexa believes these results represent an important scientific milestone: ERNA-201 demonstrated meaningful preclinical proof-of-concept in inflammatory disease, providing additional evidence that the Company’s engineered iMSC platform can be programmed to deliver therapeutically relevant factors across distinct disease settings.

With this platform validation in hand, Ernexa is concentrating its near-term capital and operational resources on what management believes represents the Company’s most immediate opportunity to create value for patients and shareholders: advancing ERNA-101 into a first-in-human clinical study in platinum-resistant ovarian cancer (PROC).

Importantly, the decision to prioritize ERNA-101 does not reflect a change in Ernexa’s conviction in ERNA-201 or its underlying biology. Rather, following the positive ERNA-201 proof-of-concept results, the Company believes it has reached an appropriate point to preserve the program while directing near-term resources toward achieving clinical validation of its platform in oncology.

"We believe the ERNA-201 results are an important validation of both the program and the broader potential of our synthetic iMSC platform," said Sanjeev Luther, President and Chief Executive Officer of Ernexa Therapeutics. "ERNA-201 did what we wanted to see at this stage: it generated an encouraging proof-of-concept signal in an established inflammatory disease model and demonstrated the potential to engineer our iMSCs for therapeutic activity beyond cancer. That success gives us the confidence to preserve the significant future opportunity represented by ERNA-201 while concentrating our resources on the milestone directly ahead of us, bringing ERNA-101 into the clinic."

Mr. Luther continued, "Our objective is bigger than advancing a single candidate. We are building a differentiated oncology company around a scalable, off-the-shelf cell therapy platform. With ERNA-101 on track for FDA IND submission before year end, we believe Ernexa is approaching a potentially defining inflection point. We intend to execute against that milestone with urgency while broadening our oncology pipeline and identifying additional cancers where our platform could make a meaningful difference."

ERNA-201: Positive Data Strengthen Validation of Ernexa’s iMSC Platform

ERNA-201 is engineered to express anti-inflammatory factors designed to target inflammation and treat autoimmune disease. In completed preliminary studies, ERNA-201 was evaluated in the CIA model, which is widely used to assess potential therapies for rheumatoid arthritis and other inflammatory joint diseases.

Mice treated with several ERNA-201 lead candidates demonstrated reductions in disease severity relative to untreated controls, including improvements in clinical arthritis scores. Ernexa believes the findings are consistent with the candidate’s intended anti-inflammatory mechanism and provide meaningful proof-of-concept support for the use of synthetic, cytokine-engineered iMSCs in inflammatory and autoimmune disease.

"Seeing disease-modifying activity from ERNA-201 in the collagen-induced arthritis model is an encouraging scientific result," said Robert H. Pierce, M.D., Chief Scientific Officer of Ernexa Therapeutics. "These findings support the underlying thesis that engineered iMSCs can serve as a versatile therapeutic delivery platform. We designed ERNA-201 to deliver anti-inflammatory factors in disease, and the preliminary activity we observed is consistent with that objective. From a scientific perspective, these data increase our confidence in the program and provide another important validation point for the broader platform."

The ERNA-201 results are particularly important to Ernexa because the program employs the same fundamental synthetic iMSC platform architecture that underpins the Company’s broader pipeline. While ERNA-201 is engineered for anti-inflammatory activity and ERNA-101 is engineered to stimulate an immune response against cancer, both programs demonstrate the potential versatility of an allogeneic, scalable cell platform that can be engineered for distinct therapeutic purposes.

Building Momentum Toward the Clinic: ERNA-101 Advancing Toward Planned IND Submission in Q4 2026

With ERNA-201 generating positive preliminary proof-of-concept data, Ernexa is prioritizing the next major milestone in the Company’s evolution: advancing ERNA-101 toward a planned IND submission for a first-in-human clinical study in platinum-resistant ovarian cancer.

ERNA-101 is Ernexa’s lead oncology cell therapy candidate, engineered to express an IL-7/IL-15 fusion cytokine designed to activate and regulate the immune system’s response to recognize and attack cancer cells.

The Company is focused on advancing ERNA-101 toward a planned IND submission in the fourth quarter of 2026. Ernexa believes achieving this milestone would represent an important step toward the Company’s transition from a preclinical development company to a clinical-stage oncology company and the generation of human clinical evidence necessary to evaluate the therapeutic potential of its platform.

Clinical data from the ERNA-101 PROC study are expected in Q4 2027, providing the Company with a series of potential clinical and operational milestones over the coming 12–18 months.

A Focused Oncology Strategy Built Around One Scalable Platform

Ernexa’s strategy extends beyond a single indication or product candidate. The Company’s proprietary platform begins with induced pluripotent stem cells (iPSCs), which are engineered and transformed into synthetic induced mesenchymal stem cells. The resulting allogeneic iMSCs are designed to provide a scalable, off-the-shelf therapeutic approach without the need for patient-specific cell harvesting.

Management believes this architecture creates the potential to engineer iMSCs with different therapeutic factors and apply the platform across multiple diseases. The positive preliminary ERNA-201 findings provide additional support for that platform thesis, while ERNA-101 now represents the Company’s most advanced opportunity to demonstrate its potential in patients.

As Ernexa moves toward the clinic, the Company intends to broaden the scope of its oncology program by evaluating additional high-need cancer indications and advancing next-generation iMSC candidates designed to build on the Company’s existing scientific and manufacturing capabilities.

The Company will prioritize opportunities based on scientific rationale, unmet patient need, commercial potential and capital efficiency, including opportunities that may be pursued internally or through strategic collaborations.

Disciplined Capital Allocation: Advancing the Nearest Value-Creating Milestones

Following the encouraging ERNA-201 results, Ernexa has elected to pause additional preclinical development expenditures on ERNA-201 at this time and preserve the program while concentrating near-term resources on ERNA-101 and the expansion of its oncology pipeline.

The decision is a capital allocation priority rather than a scientific decision to discontinue ERNA-201. The Company believes the positive preliminary results provide a foundation from which ERNA-201 could potentially be advanced in the future as resources, strategic priorities and development opportunities warrant.

"We have achieved an important objective with ERNA-201 by generating encouraging proof-of-concept data," Luther said. "The question for us now is not whether we believe in the program. We do. The question is where each incremental dollar and each hour of our team’s effort can have the greatest impact today. With ERNA-101 approaching the clinic, we believe concentrating resources behind first-patient dosing and expanding our oncology opportunity is the most disciplined path forward."

"We are preserving ERNA-201’s future potential while accelerating the program that is closest to human clinical validation. We believe that the combination of scientific validation, disciplined execution and a clear path into the clinic positions Ernexa for an important next chapter."

Multiple Potential Catalysts Ahead

Over the coming 12–18 months, Ernexa intends to:

Submit ERNA-101 IND in Q4 2026 for a first-in-human clinical study in platinum-resistant ovarian cancer;
Generate clinical data from the ERNA-101 PROC program, currently expected in Q4 2027;
Continue generating translational and preclinical evidence designed to strengthen validation of the synthetic iMSC platform;
Evaluate opportunities to expand the platform into additional high-need oncology indications;
Advance next-generation synthetic iMSC oncology candidates;
Explore strategic collaborations that could accelerate or broaden the application of Ernexa’s technology; and
Maintain disciplined capital allocation focused on milestones management believes can create the greatest value for patients and shareholders.
"We believe we are entering one of the most consequential periods in Ernexa’s development," Luther concluded. "ERNA-201 has provided encouraging evidence that our engineering approach can translate into therapeutic activity in a well-established disease model. ERNA-101 is approaching the clinic. And behind it is a platform we believe can be engineered and deployed against multiple high-need cancers. Our focus now is execution, reaching the clinic, generating human data and building a broader oncology pipeline capable of creating sustainable value."

(Press release, Ernexa Therapeutics, SEP 15, 2026, View Source [SID1234670884])

KORTUC Achieves Target Enrollment in Registrational Breast Cancer Trial as Three Global Clinical Programs Advance

On September 15, 2026 KORTUC INC., a clinical-stage oncology biotechnology company developing an investigational intratumoral radiosensitizer, reported that it has achieved its target enrollment of 184 patients in its registrational, randomized Phase II breast cancer trial. The study is now moving toward patient follow-up, data analysis, and an efficacy readout targeted for 2027. The news comes following a Series B round and broader company update: KORTUC now has three clinical studies advancing in breast, cervical and rectal cancer.

Together, they reflect a growing international development program – including a U.S. academic collaboration with the University of California San Diego, a United Kingdom-led breast cancer trial and a newly authorized France-based rectal cancer study, with additional trial sites in India and Thailand.

KRC-01, a proprietary intratumoral formulation based on hydrogen peroxide and sodium hyaluronate, addresses hypoxia – or lack of oxygen – a problem for over 50% of solid tumors. It is designed to raise oxygen levels within tumors and inactivate antioxidative enzymes that contribute to radioresistance. Sodium hyaluronate slows the decomposition of hydrogen peroxide, helping the formulation remain within the tumor as radiation treatment is delivered.

Three clinical programs advancing globally

Breast cancer trial reaches 184-patient enrollment target: The breast cancer trial is led by The Institute of Cancer Research in the United Kingdom. The study’s primary endpoint is complete response – no detectable tumor on MRI after treatment, a particularly meaningful outcome for patients with locally advanced or recurrent disease. It compares radiotherapy alone with radiotherapy plus KRC-01 in patients with primary or recurrent locally advanced breast cancer. The study is being conducted through six centers in the United Kingdom and four centers in India (NCT03946202).

"Reaching our 184-patient enrollment target is a defining milestone for KORTUC," said Kazu Matsuda, CEO of KORTUC. "It moves our lead program beyond target recruitment and toward data readout, while our cervical and rectal studies expand the evaluation of KRC-01 into two additional cancers. Through clinical collaborations in the United States, United Kingdom and France, we are building the global evidence needed to assess KRC-01 across tumors where hypoxia can limit radiation response."

Cervical cancer – global study with U.S. academic collaboration: KORTUC’s ongoing global Phase 1/2 study, conducted in partnership with the University of California San Diego, is evaluating KRC-01 with standard chemoradiotherapy in 70 patients with untreated, locally advanced cervical cancer. Following first enrollment in March 2026, the study is progressing through a 10-patient safety and dose-schedule phase before a 60-patient randomized phase comparing standard treatment with or without KRC-01. The randomized phase will primarily evaluate progression-free survival (PFS), while also assessing overall survival (OS) and quality of life, examining whether adding KRC-01 can translate into meaningful longer-term benefits for patients. Registry sites are listed in Thailand, India and the United Kingdom (NCT05570422).

Rectal cancer – France: The Phase II K-BOOST clinical trial has received authorization to proceed in France and is expected to begin enrollment following site activation. Sponsored by Gustave Roussy, the study will enroll 24 adults with non-metastatic, high-risk stage III rectal cancer at Gustave Roussy and Institut Bergonié. Participants will receive eight intratumoral KRC-01 injections alongside standard long-course chemoradiotherapy, followed by consolidation chemotherapy. The primary endpoint is the clinical complete response rate at week 24. The study will explore whether adding KRC-01 can support an organ-preservation pathway for patients while retaining surgery as an established treatment option. K-BOOST is registered under EU Clinical Trial number 2025-524378-40-00 and ClinicalTrials.gov identifier NCT07622017.

KRC-01 is an investigational product and has not been approved for commercial use. For more information about KORTUC and its clinical development program, visit KORTUC.com.

(Press release, KORTUC, SEP 15, 2026, View Source [SID1234670883])

Elevar Therapeutics Submits Marketing Authorization Application to European Medicines Agency for Lirafugratinib as a Treatment for Patients with Advanced/Metastatic Cholangiocarcinoma (CCA) Harboring FGFR2 Fusions or Rearrangements

On September 15, 2026 Elevar Therapeutics, Inc., a majority-owned subsidiary of HLB Co., Ltd. and a fully integrated biopharmaceutical company dedicated to elevating treatment experiences and outcomes for cancer patients, reported the submission of a marketing authorization application to the European Medicines Agency (EMA) for lirafugratinib as a second-line treatment for patients with advanced/metastatic cholangiocarcinoma (CCA) harboring FGFR2 fusions or rearrangements.

The application is based on results of the Phase 1/2 ReFocus study, of which the primary endpoint is objective response rate (ORR). In advanced/metastatic FGFR2 fusion+ CCA patients after receiving a prior line of systemic therapy, lirafugratinib achieved an ORR of 46.5% as assessed by independent review committee. The median duration of response, a key secondary endpoint, was 11.8 months. Median progression-free survival was 11.3 months, while median overall survival was 22.8 months.

"Lirafugratinib is a novel drug candidate that has demonstrated meaningful clinical activity and a differentiated safety profile consistent with its high selectivity for FGFR2 and sustained inhibitory mechanism," said Dong-Gun Kim, chief executive officer of Elevar. "We will continue to advance the regulatory processes in the United States and European Union while broadening the therapeutic potential of lirafugratinib across multiple solid tumors through our tumor-agnostic clinical development program."

Lirafugratinib is an irreversible inhibitor that selectively targets FGFR2, differentiating it from existing pan-FGFR inhibitors that broadly inhibit FGFR1, FGFR3, and/or FGFR4 in addition to FGFR2. Consistent with its high selectivity, the rates of hyperphosphatemia and diarrhea – adverse events commonly associated with non-selective FGFR inhibitors – were 20.7% and 21.6%, respectively.

Lirafugratinib received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) in 2022 and Breakthrough Therapy Designation in 2023. Elevar submitted a New Drug Application for lirafugratinib to the FDA in January 2026, and the application was granted Priority Review. The FDA is currently reviewing the application, with a Prescription Drug User Fee Act target action date of Sept. 25, 2026.

Elevar is also conducting ReFocus202, a global Phase 2 study evaluating lirafugratinib in patients with various solid tumors harboring FGFR2 fusions or rearrangements. Patient enrollment and dosing are currently underway at clinical sites globally, including in the EU, U.K., Korea and U.S. Through this program, Elevar seeks to expand the therapeutic potential of lirafugratinib beyond CCA to a broader range of solid tumors with a FGFR2 fusion or rearrangement.

About Lirafugratinib

Lirafugratinib (aka RLY-4008) is a potent, selective and oral small molecule inhibitor of FGFR2, a receptor tyrosine kinase that is frequently altered in certain cancers. FGFR2 is one of four members of the FGFR family, a set of closely related proteins with highly similar protein sequences and properties. Lirafugratinib is currently being evaluated in a clinical trial to enroll additional patients with previously treated, advanced or metastatic solid tumors other than CCA harboring FGFR2 fusion or rearrangement, who have not been treated with prior FGFR inhibitors. Elevar has an exclusive license to lirafugratinib from Relay Therapeutics, Inc. for commercialization worldwide.

(Press release, Elevar Therapeutics, SEP 15, 2026, View Source [SID1234670882])

Genexine’s GX-BP1 (SOX2 bioPROTAC) Suppresses Tumor Regrowth despite Osimertinib and Dato-DXd Combination and Achieves 6/6 Complete Responses in Triple Combination

On September 15, 2026 Genexine, Inc. (KOSDAQ: 095700) reported that Jaehyun Choi, Ph.D., CEO and Head of R&D, presented new preclinical data for GX-BP1, the company’s SOX2-targeting bioPROTAC, at the 2026 World Conference on Lung Cancer (WCLC) in Seoul on September 13.

The mini oral presentation, titled ‘Lung-Targeted mRNA-LNP Delivery of SOX2 bioPROTAC: A Comprehensive Strategy for LUAD, LUSC, and SCLC in Monotherapy and Combination Settings’ highlighted the potential of GX-BP1 to overcome treatment resistance and suppress tumor regrowth when combined with standard lung cancer therapies, including the EGFR-targeted therapy osimertinib and the TROP2 antibody-drug conjugate datopotamab deruxtecan (Dato-DXd).

In an HCC827 EGFR-mutant lung cancer xenograft model, osimertinib initially produced strong tumor control, but tumors began to regrow after treatment ended at day 20. The combination of osimertinib and Dato-DXd delayed regrowth, but tumors eventually progressed from day 40 and showed significant volume after day 70.

In contrast, osimertinib plus GX-BP1 combination therapy maintained durable tumor control for more than 100 days, with no substantial tumor regrowth observed during the study period. Most notably, the triple combination of GX-BP1, osimertinib and Dato-DXd achieved complete responses in all six animals (6/6 CR) with no regrowth observed.

Genexine also presented data from a Dato-DXd-resistant tumor model. GX-BP1 alone achieved 92.3% tumor growth inhibition (TGI), while the combination of GX-BP1 and Dato-DXd achieved 115.3% TGI, indicating tumor regression from baseline despite resistance to the ADC.

Targeting SOX2 to Address Recurrence and Drug Resistance

Genexine believes the antitumor activity of GX-BP1 is driven by degradation of SOX2, a transcription factor associated with cancer stemness, treatment resistance, recurrence and metastasis.

In EGFR-mutant lung cancer cells, treatment with osimertinib increased SOX2 expression in a dose-dependent manner. Similar increases were observed following treatment with other EGFR-targeted therapies, including lazertinib and amivantamab.

Osimertinib treatment also increased the expression of genes associated with drug efflux pumps, a resistance mechanism that reduces intracellular drug exposure by transporting therapeutic agents or cytotoxic compounds out of cancer cells. Such mechanisms may also reduce intracellular exposure to cytotoxic payloads delivered by ADCs.

By degrading SOX2, GX-BP1 is designed to target cancer stem cells and drug-persistent cancer cells while reducing mechanisms associated with treatment resistance.

"Our goal with GX-BP1 is to develop it as a combination backbone molecule with any standard therapies for lung cancer as an anti-resistant anti-cancer therapy" said Dr. Choi. "The WCLC data support the potential of GX-BP1 to improve the durability of response when combined with EGFR-targeted therapies and ADCs."

Dr. Choi added, "The observed effect on drug-efflux mechanisms generated significant interest during our WCLC presentation. We plan to further develop GX-BP1 in combination with established lung cancer therapies and actively pursue global licensing opportunities with companies developing EGFR-targeted agents and ADCs."

(Press release, Genexine, SEP 15, 2026, View Source [SID1234670881])