On November 17, 2025 Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company pursuing development of targeted therapies for oncology, reported the completion of the submission of its New Drug Application ("NDA") to the U.S. Food and Drug Administration ("FDA") for gedatolisib in hormone receptor positive ("HR+"), human epidermal growth factor receptor 2 negative ("HER2-"), advanced breast cancer ("ABC"). The NDA was submitted under the FDA’s Real-Time Oncology Review ("RTOR") program, which is intended to facilitate shorter regulatory review periods. Gedatolisib previously received both Breakthrough Therapy and Fast Track designations based on promising preliminary clinical data. The submission is based on clinical data from the PIK3CA wild-type cohort of the Phase 3 VIKTORIA-1 clinical trial.

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"This NDA submission is an important milestone, and it brings gedatolisib one step closer to becoming available for patients with HR+/HER2- advanced breast cancer," said Brian Sullivan, CEO and co-founder of Celcuity. "We look forward to working with the FDA during the NDA review process. We believe the unprecedented efficacy results and overall safety profile of the gedatolisib regimens are potentially practice changing for patients with HR+/HER2- advanced breast cancer."

The NDA submission is based on the positive clinical results for the PIK3CA wild-type cohort of the Phase 3 VIKTORIA-1 trial. The efficacy results established several new milestones in the history of drug development for HR+/HER2- ABC. The gedatolisib-triplet (gedatolisib, fulvestrant and palbociclib) reduced the risk of disease progression or death by 76% compared to fulvestrant based on a hazard ratio of 0.24. The median progression-free survival ("PFS") was 9.3 months with the gedatolisib-triplet versus 2.0 months with fulvestrant, an incremental improvement of 7.3 months. The gedatolisib-doublet (gedatolisib and fulvestrant) reduced the risk of disease progression or death by 67% compared to fulvestrant based on a hazard ratio of 0.33. The median PFS was 7.4 months with the gedatolisib-doublet versus 2.0 months with fulvestrant, an incremental improvement of 5.4 months.

Gedatolisib

Gedatolisib is an investigational, multi-target PI3K/AKT/mTOR ("PAM") inhibitor that potently targets all four Class I PI3K isoforms, mTORC1, and mTORC2 to induce comprehensive blockade of the PAM pathway.1,2,3 As a multi-target PAM inhibitor, gedatolisib’s mechanism of action is highly differentiated from currently approved single-target inhibitors of the PAM pathway.2 Inhibition of only a single PAM component results in cross-activation of the uninhibited components, which limits the suppression of PAM pathway activity. Gedatolisib’s comprehensive PAM pathway inhibition enables full suppression of the PAM pathway by minimizing the adaptive cross-activation that occurs with single-target inhibitors. Unlike single-target inhibitors of the PAM pathway, gedatolisib has demonstrated comparable potency and cytotoxicity in PIK3CA-mutant and wild-type breast tumor cells in nonclinical studies and early clinical data.

About RTOR

The FDA established the RTOR program to facilitate a more efficient review process for drugs to ensure that safe and effective treatments are available to patients as early as possible, while improving review quality and engaging in early iterative communication with the applicant. To be considered for RTOR, submissions should demonstrate the following: 1) clinical evidence from adequate and well-controlled investigation that indicates the drug may demonstrate substantial improvement on a clinically relevant endpoint over available therapies; 2) easily interpreted clinical trial endpoints; and 3) no aspect of the submission is likely to require a longer review time. Additional information about RTOR can be found at: View Source

(Press release, Celcuity, NOV 17, 2025, View Source [SID1234660015])

On November 17, 2025 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical development stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, reported that the U.S. Patent and Trademark Office (USPTO) has issued a Notice of Allowance for a key patent, entitled, ‘Deoxy-Cytidine or Uridine Derivatives for Use in Cancer Therapies’ (Pat. Appl. Ser. No. 18/602,969), which covers the use of GLIX1 for treating cancer types in which cytidine deaminase (CDA) is not over-expressed beyond a specific threshold. Corresponding patent applications are pending worldwide. The patent further broadens and strengthens GLIX1’s patent protection for the treatment of cancer until 2040, with a possible patent-term extension of up to five years.

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It is estimated that over 90% of all cancers do not overexpress CDA beyond the specific threshold, reflecting the importance of this additional intellectual property as BioLineRx expands development of GLIX1 beyond glioblastoma, its initial indication, into other cancer types, once safety and dosing in glioblastoma are established.

"This Notice of Allowance from the USPTO is a critical addition to GLIX1’s intellectual property estate, as it protects our opportunity to evaluate this exciting molecule across the majority of cancers," stated Philip Serlin, Chief Executive Officer of BioLineRx. "As we prepare to initiate a first-in-human study of GLIX1 in patients with glioblastoma in the first quarter of 2026, we are in parallel advancing pre-clinical studies in other cancer models. We believe GLIX1, with its unique mechanism of action that targets the DNA damage response in cancer cells, has the potential to offer new hope to patients suffering from a broad range of difficult-to-treat cancers, and we are eager to commence clinical trials early next year."

GLIX1 is covered by a comprehensive portfolio of patents that are both issued and pending. In addition to the allowed U.S. patent referenced above and corresponding patents issued and pending worldwide, GLIX1 is covered by additional issued or pending patents, which broaden the coverage for its use in treating cancer as both monotherapy and in combination with established anti-cancer agents:

GLIX1 for use in treating cancer of the central nervous system, such as glioblastoma, is covered by patents issued in the US, Europe and 13 other countries. The patents are valid until at least 2040 (with a possible patent term extension of up to five years).
GLIX1, in combination with PARP inhibitors, for use in treating homologous recombination (HR) proficient cancers, which represent the majority of cancers, is covered by a pending international patent application. Corresponding national-phase patents, if granted, will be valid until at least 2044 (with a possible patent term extension of up to five years).

(Press release, BioLineRx, NOV 17, 2025, View Source [SID1234660014])

On November 17, 2025 Artios Pharma Limited ("Artios"), a biopharmaceutical company committed to realizing the therapeutic power of targeting the DNA damage response ("DDR") in cancer, reported the successful close of an oversubscribed $115 million Series D financing. The round was co-led by founding investor SV Health Investors and new investor RA Capital Management, with participation from new investor Janus Henderson Investors and broad support from Artios’ existing investors.

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The Series D proceeds will expand the clinical evaluation of Artios’ lead program, alnodesertib, to enroll additional ATM-negative1 patients in each of second-line pancreatic cancer and third-line colorectal cancer, for which the program was recently granted U.S. FDA Fast Track Designation. At the AACR (Free AACR Whitepaper) meeting in April 2025, Artios reported that alnodesertib, in combination with low-dose irinotecan, demonstrated a 50% confirmed overall response rate in patients with ATM-negative solid tumors at the recommended Phase 2 dose in the STELLA Phase 1/2a trial. There are currently no approved therapies specifically for patients whose tumors harbor ATM-deficiency, a population where alnodesertib has demonstrated durable responses across eight different solid tumors.

The proceeds from the financing will also be used to initiate a Phase 2 randomized clinical trial for Artios’ second potential first-in-class candidate, ART6043, in patients with BRCA-mutant HER2-negative breast cancer who are eligible to receive a PARP inhibitor. The DNA polymerase Theta (Polθ) inhibitor, ART6043, demonstrated an attractive tolerability profile, expected PK/PD activity, and promising clinical signals in data from a Phase 1/2a study presented at the ESMO (Free ESMO Whitepaper) Congress in September 2025. The company is also advancing a first-in-class and highly differentiated DDR inhibitor-Antibody Drug Conjugate (DDRi-ADC) program and expects to name a lead candidate in Q1 2026.

"This Series D accelerates our potential path to registration for both alnodesertib and ART6043, broadening development for the next generation of DNA damage response (DDR) therapeutics to indications among the highest of unmet need across pancreatic, colorectal, and breast cancers, where median survival is often measured in months," said Mike Andriole, Chief Executive Officer of Artios. "As we address these indications and prepare for others, I would like to thank our existing investors, led by SV Health, for their ongoing support, and also our new investors, RA Capital Management and Janus Henderson Investors, for joining our mission to bring these potential medicines to patients as quickly as possible."

Nikola Trbovic, Managing Partner, SV Health Investors, added, "We are thrilled to have supported Artios’ evolution, from an early-stage DDR pioneer when we founded the company to the established company it has become, distinguished by a promising and differentiated pipeline. We look forward to continuing to do so as it deploys the Series D proceeds to drive late-stage development of alnodesertib as well as its pipeline. This financing, and the recent appointment of Mike Andriole as CEO, are exciting steps in Artios’ continued growth and its transition toward becoming a commercially oriented organization."

Jake Simson, Partner, RA Capital Management, commented, "We are excited to co-lead this financing round to advance the next generation of DNA damage response therapeutics. Artios’ differentiated clinical programs, alnodesertib and ART6043, together have the potential to meaningfully expand the impact of DDR-targeted therapies. The rate and durability of responses observed to date for alnodesertib across a range of solid tumors and the early clinical results with ART6043 underscore the strength of Artios’ approach and ability to deliver novel, potentially first-in-class treatments for patients while building significant long-term value."

The investors who supported the Series D round include Andera Partners, Avidity Partners, EQT Life Sciences, Invus, IP Group plc, Janus Henderson Investors, M Ventures, Novartis Venture Fund, Omega Funds, Pfizer Ventures, Piper Heartland, RA Capital Management, Sofinnova Partners, Schroders Capital, and SV Health Investors.

(Press release, Artios Pharma, NOV 17, 2025, View Source [SID1234660013])

On November 17, 2025 Privo Technologies reported results from the CLN-004 Phase 2/3 (Cohort 1) trial evaluating PRV111, a nanoengineered, cisplatin-releasing patch for the treatment of non-invasive oral cancer and high-grade dysplasia (HGD). These conditions are common, recur frequently, and typically require repeated surgical excision, which can impair speech, swallowing, and appearance.

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In this trial, PRV111 delivered a 92% pathologic CR rate, eliminated the need for surgery in all patients, and demonstrated durable local control. Manijeh Goldberg, PhD, MBA, Founder and CEO of Privo Technologies, commented:
"The complete response rate achieved with PRV111 is among the highest reported for localized therapies in this setting. Patients typically face repeated surgical resections, which can lead to fibrosis, scar formation, and anatomic distortion that can permanently affect speech and other functionality. This non-surgical patch not only cleared the tumors but preserved native tissue function. These results reinforce PRV111’s potential to redefine the standard of care for oral precancer and early carcinoma in situs. We are one step closer to providing patients with an office-based, non-surgical alternative to repeated operative procedures."

The CLN-004 study builds upon Privo’s earlier clinical experience with PRV111 (CLN-001), which showed promising local tumor responses with no systemic toxicity in a first-in-human setting. The results of that earlier trial were published in Nature Communications and highlighted by Forbes for their innovative approach to localized cancer drug delivery.

Detailed Results:
Twelve patients with biopsy-confirmed non-invasive oral cancers or high-grade dysplasia received a single cycle of PRV111. Eleven patients achieved centrally confirmed pathologic CRs; the twelfth patient was down staged to low-grade dysplasia, eliminating the need for surgery. All treated lesions resolved, and mucosal surfaces regenerated without scarring or distortion. At a median follow-up of ~6 months, no recurrences were observed; several patients have maintained responses beyond six months, and the longest ongoing response now ~12 months. There were no systemic toxicities, dose-limiting toxicities, or treatment-related serious adverse events. Treatment-related events were primarily mild, transient oral symptoms.

Transforming the Treatment Paradigm:
Current management of non-invasive oral cancer and HGD relies on surgical excision, which is associated with high recurrence and morbidity. PRV111 is designed to deliver high concentrations of cisplatin directly into dysplastic tissue through a brief, office-based procedure, avoiding systemic side effects associated with traditional chemotherapy. This topical approach represents the first non-surgical alternative in development focused on treating this class of disease.

Next Steps:
Privo Technologies is initiating a registrational study of PRV111 in non-invasive oral cancers and high-grade dysplasias. The company will also explore PRV111 in other early malignant and premalignant lesions where non-surgical alternatives could provide significant benefits.

About PRV111:
PRV111 is a nanoengineered, polymeric topical patch designed to deliver high-dose cisplatin directly into oral lesions and adjacent mucosa while minimizing systemic exposure. The patch adheres to the lesion, achieves rapid drug permeation into dysplastic tissue and regional lymphatics, and is removed after treatment.

(Press release, Privo Technologies, NOV 17, 2025, View Source [SID1234660006])

On November 17, 2025 Syntara Limited (ASX: SNT), a clinical-stage drug development company, reported that it has received a positive opinion on the submission of Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) for its advanced clinical asset amsulostat (SNT-5505) for the treatment of myelofibrosis (MF).

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An ODD grant will provide numerous incentives for Syntara in the European Union (EU) as it develops amsulostat, including ten years market exclusivity upon approval, clinical trial protocol assistance, access to the centralised authorisation procedure in Europe, and certain fee reductions.

Amsulostat already has ODD in the US from the Food & Drug Administration (FDA) for the treatment of MF.

Myelofibrosis is a disorder in which normal bone marrow tissue is gradually replaced with a fibrous scar-like material. Over time, this leads to progressive bone marrow failure. It can occur at any age but is usually diagnosed later in life, between the ages of 60 and 70 years, with its cause remaining largely unknown.

Syntara recently announced positive top-line Phase 2a data for amsulostat in MF, with patients sub optimally controlled on ruxolitinib with 73% achieving at least a 50% reduction in total symptom score, and nearly half showing meaningful spleen volume reduction after a year of treatment. This sustained improvement in clinical measures of efficacy is backed up by excellent tolerability and no treatment-related adverse events.

Syntara has received feedback from the FDA on a recommended pathway for further development, and is progressing a design for the next trial of amsulostat.

Syntara CEO Gary Phillips commented: "Receipt of this positive opinion for Orphan Drug Desgination in the EU comes after detailed review of the amsulostat pre-clinical and clinical dossier by the EMA, providing further validation of the potential that the drug has to benefit myelofibrosis patients. We continue to engage with our advisors and regulatory authorities to design a clinical trial that leverages the product’s strong differentiating features as a well-tolerated and effective therapy. Our goal is to ensure the study design is compelling for investors and strategic partners by clearly demonstrating how amsulostat can positively impact the current standard of care."

(Press release, Syntara, NOV 17, 2025, https://mcusercontent.com/add2e2fa70ec3d0eeaf2a93cc/files/42cc5d05-d389-5ba0-606c-034c87587de6/03023812.pdf [SID1234660003])