On September 11, 2025 Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company dedicated to unlocking the power of a patient’s own biology for the treatment of rare diseases, reported that it will host a virtual Investor Day on Thursday, October 2, 2025, from 10:00 a.m. and 12:00 p.m. Eastern Time (Press release, Quince Therapeutics, SEP 11, 2025, View Source [SID1234655936]). Quince’s virtual Investor Day will feature presentations from the company’s leadership team who will share the latest clinical development and corporate updates including:

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Quince Overview – Learn about the company’s pioneering Autologous Intracellular Drug Encapsulation (AIDE) technology designed to chronically deliver corticosteroids without toxicity and the potential to transform treatment paradigms across multiple rare disease indications where corticosteroids are the standard of care.
Technology Deep Dive – A look at the company’s proprietary eDSP System, which is a unique drug/device combination that uses an automated process designed to encapsulate dexamethasone sodium phosphate (eDSP) into a patient’s own red blood cells to allow for the chronic, monthly administration of corticosteroids without toxicity.
Lead Indication: Ataxia-Telangiectasia (A-T) – Insights into this devastating pediatric rare disease, encouraging prior ATTeST study results, and the company’s fully enrolled pivotal Phase 3 NEAT (Neurological Effects of eDSP on Subjects with A-T; NCT06193200/IEDAT-04-2022) clinical trial with topline results expected in the first quarter of 2026.
Mechanism of Action – Learn about multiple synergistic mechanisms of action that support eDSP’s efficacy and disease modifying activity while mitigating corticosteroid toxicity, and how transcriptomic profiling reveals novel insights and potential biomarkers in A-T.
Regulatory Overview – Discussion of the company’s Special Protocol Assessment (SPA) agreement in place with the U.S. Food and Drug Administration (FDA), orphan drug and Fast Track designations for the treatment of A-T, and the anticipated regulatory approval pathway for eDSP, assuming positive NEAT study results.
Commercial Development – Insights into the company’s attractive commercial opportunity and launch preparedness planning for eDSP, including details of its recently announced strategic relationship with Option Care Health.
Driving Value Creation – A look at the company’s strong competitive positioning, significant opportunity to quickly expand its development pipeline into additional high-value, rare disease indications like Duchenne muscular dystrophy, and its existing cash runway which provides funding through topline results and into the second quarter of 2026.
Q&A Session – Delve into the details during a Q&A session with Quince’s leadership team following the conclusion of formal presentations.
To register for this webinar, please click here. A live webcast of the presentation will be accessible on Quince’s Events page under the News & Events heading of the company’s Investor Relations website at ir.quincetx.com. An archive of the webcast will be available shortly following the end of the live event.

On September 11, 2025 Kazia Therapeutics Limited (NASDAQ: KZIA), an oncology-focused drug development company, reported new findings from a collaborative research program led by Professor Sudha Rao at QIMR Berghofer (Press release, Kazia Therapeutics, SEP 11, 2025, View Source [SID1234655934]).

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In this ex vivo study, blood samples from Stage IV HER2-positive metastatic breast cancer (mBC) patients were profiled to evaluate the effect of paxalisib, Kazia’s investigational PI3K–mTOR inhibitor, on metastatic burden. Paxalisib monotherapy demonstrated a statistically significant reduction in single circulating tumor cells and achieved a complete (100%) disruption of circulating tumor cell (CTC) clusters containing three or more cells."

Key Points
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HER2-positive breast cancer accounts for 15–20% of cases and remains a clinical challenge despite the transformative impact of HER2-targeted therapies, with many patients experiencing resistance, recurrence, or metastasis.

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Immunotherapy has demonstrated success across several solid tumors but has shown limited efficacy in HER2-positive breast cancer, underscoring the need for new therapeutic approaches.

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In this study, liquid biopsy profiling of Stage IV patients revealed that paxalisib treatment effectively disrupted CTCs and CTC clusters, which are considered biomarkers of aggressive disease and metastasis.

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Immunofluorescence analyses showed that paxalisib-treated blood samples from HER2-positive mBC patients achieved complete disruption of highly metastatic CTC clusters (≥3 cells).

"This monotherapy ex-vivo result extends our understanding of paxalisib’s potential beyond triple-negative breast cancer into HER2-positive disease," said Dr. John Friend, CEO of Kazia Therapeutics. "The ability to disrupt circulating tumor cell clusters, which are strongly associated with metastasis and poor prognosis, represents a transformative therapeutic avenue. We are particularly excited by the precision medicine aspect of this work, which leverages biomarkers to both track metastatic burden and guide therapeutic decisions. This work underscores Kazia’s commitment to expanding paxalisib’s utility across multiple subtypes of advanced breast cancer, addressing high unmet need in patients with limited options."

These findings complement Kazia’s ongoing Phase 1b trial in Stage IV triple-negative breast cancer (TNBC), where initial patient data announced in July 2025 demonstrated significant reductions in circulating tumor cells and clusters, underscoring the broader potential of paxalisib to address metastatic progression across multiple breast cancer subtypes.

Next Steps

Detailed datasets encompassing metastatic signatures and disrupted progenitor populations in Stage IV HER2-positive breast cancer have been submitted for presentation at an upcoming global oncology meeting in 2025.

On September 11, 2025 Kairos Pharma, Ltd. (NYSE American: KAPA), a clinical-stage biopharmaceutical company focused on innovative cancer therapeutics, reported that it will host a premier KOL event on Thursday, Sept. 18th at 5 p.m. ET / 2 p.m. PT to discuss diverse perspectives on the Company’s interim efficacy results from a Phase 2 trial of its lead candidate, ENV105, in treating advanced prostate cancer patients (Press release, Kairos Pharma, SEP 11, 2025, View Source [SID1234655933]). Registration is required to participate in the webcast. Interested participants can sign-up to receive the webcast link here.

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"The safety results of the trial are an important catalyst as we prepare for the announcement of our interim efficacy results in treating prostate cancer patients. This predetermined efficacy analysis occurs four months after start of combination therapy of ENV105 and apalutamide in the last safety lead-in patient. We hope to lay out the primary benefits of our compound, and to clearly demonstrate the clinical need filled by ENV105," said Dr. John Yu, CEO of Kairos Pharma. "This distinguished panel of experts will provide participants with a renewed understanding of the importance of these data and the role ENV105 can have in targeting cancer drug resistance."

Speakers in the KOL event include Dr. Neil Bhowmick, President and Chief Scientific Officer at Kairos Pharma; Dr. Umang Swami, Associate Professor in the Division of Oncology, Department of Internal Medicine at the Huntsman Cancer Institute; Dr. Richard Lee, Clinical Co-Director, The Claire and John Bertucci Center for Genitourinary Cancers at Massachusetts General Hospital, Harvard Medical School, and Dr. Edwin Posadas, Director of the Experimental Therapeutics Program and the Medical Director of the Center for Uro-Oncology Research Excellence at the Samuel Oschin Comprehensive Cancer Institute.

The interim safety analysis of the trial, announced in July of this year, demonstrated that ENV105, a first-in-class CD105 antagonist, was well tolerated when combined with standard of care hormone therapy, apalutamide, from the first 10 enrolled patients. Thus far, there have been no dose-limiting toxicities or unexpected adverse events reported to date. In addition, the treatment-related side effects were manageable with standard supportive care. Notably, no Grade 3 or 4 toxicities were observed.

With one million men in the US diagnosed with prostate cancer each year, and millions more worldwide, the development of resistance to current hormone therapies is a growing unmet need with an increasingly aging population. Castration-resistant prostate cancer refers to tumors that grow despite receiving hormone blocking agents. Treatment options remain limited after hormone therapies fail. Kairos Pharma seeks to provide a safe and effective alternative for these patients with ENV-105.

On September 11, 2025 Ferring Pharmaceuticals Co., Ltd. reported that the PMDA has accepted the NDA for nadofaragene firadenovec for review, following submission on August 27th, 2025 (Press release, Ferring, SEP 11, 2025, View Source [SID1234655932]). This non-replicating gene therapy, administered intravesically, offers patients with NMIBC a bladder-sparing treatment option. Nadofaragene firadenovec’s quarterly dosing eliminates the burden of frequent treatments, while delivering a non-chemotherapy mechanism of action through interferon gene therapy. The NDA acceptance further highlights Ferring’s ongoing commitment to establish the new standard of care for high-risk BCG- unresponsive NMIBC.

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Professor Keiji Inoue, M.D., Ph.D., Department of Urology, Kochi Medical School, stated: "Nadofaragene firadenovec represents an option for those who failed NMIBC treatment. As the first choice after BCG failure, this bladder sparing gene therapy offers patients a non-chemotherapy option that transforms their own bladder cells into interferon-producing factories. The 75% complete response rate achieved with convenient quarterly dosing provides hope for patients who previously faced limited treatment options."

The NDA for nadofaragene firadenovec is based on results from a Phase 3 trial conducted in Japan.1 The primary outcome of a complete response (CR) rate of 75% at 3 months, following a single dose in 20 high-risk Japanese patients with carcinoma in situ (CIS), with or without concomitant high-grade Ta or T1 papillary lesions, was presented at the 112th Annual Meeting of the Japanese Urological Association (JUA, April 17-19, 2025, Fukuoka). All treatment-related adverse events were limited to Grade 1 (84.2%) or Grade 2 (15.8%), with zero Grade 3, 4, or 5 adverse events reported – confirming the therapy’s favourable safety and tolerability profile.1

These results are consistent with independent US real-world data presented by the Mayo Clinic, which demonstrated 79% complete response rate.2 The results from the Mayo Clinic were achieved following a single quarterly dosing, representing clinical and convenience advantages over existing therapies, without requiring re-induction protocols.2 Patients who achieved complete response continued quarterly maintenance dosing.

Joern Jakobsen, M.D., Ph.D., Vice President and Head of Global Research and Medical for Uro-Oncology and Urology, Ferring Pharmaceuticals stated "Traditionally, when BCG therapy proved insufficient, patients had no choice but to undergo highly invasive treatments such as radical cystectomy or risk cancer progression. Nadofaragene firadenovec is expected to offer a new bladder-sparing treatment option. At Ferring, we are committed to addressing the unmet needs in bladder cancer treatment by providing urologists with critical insights that enable effective, personalized, and groundbreaking therapies."

"Our ambition is to establish nadofaragene firadenovec as the new standard of care and the backbone therapy for NMIBC treatment," said Bipin Dalmia, Global Head, Uro-Oncology & Urology Franchise. "High-risk NMIBC patients who no longer respond to BCG have endured decades of little progress and currently face bladder removal as their primary option. This PMDA acceptance validates our strategic commitment to bring this treatment to Japanese patients."

About nadofaragene firadenovec
Nadofaragene firadenovec represents the first and only FDA-approved intravesical non-replicating gene therapy for adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumours.

As the first choice therapy following BCG failure, this non-chemotherapy approach utilises a non-replicating adenovirus vector-based therapy containing the interferon alfa-2b gene. Administered locally as convenient monotherapy by catheter directly into the bladder once every three months only, the mechanism transforms bladder wall cells into interferon microfactories, creating high and transient local expression of interferon alfa-2b protein – amplifying the body’s natural cancer-fighting capabilities through gene therapy rather than traditional chemotherapy approaches.

This therapeutic approach has been investigated through comprehensive clinical evaluation including 157 patients in the US with high-risk BCG-unresponsive NMIBC who demonstrated inadequate BCG response (full inclusion criteria published on clinicaltrials.gov: NCT02773849). 5-year follow-up data confirmed 80% overall survival rate and 49% cystectomy-free survival rate in adult patients with high-risk BCG-unresponsive NMIBC with CIS with or without papillary tumours (±Ta/T1), and in patients with high-grade Ta/T1 without CIS. Long-term safety profile maintained with most treatment emergent AEs remaining transient Grade 1 or 2 (66% of all patients studied), and 4% experiencing Grade 3 AEs3.

In the real world setting, between November 2023 and October 2024, 45 patients across three Mayo Clinic locations in the US were treated with nadofaragene firadenovec for BCG-unresponsive NMIBC. Out of 45 treated patients, 29 were included in the analysis.2 After a median follow-up of 8.2 months: 72% of patients showed a complete response or were free from high-grade recurrence at 3 months. 62% maintained this response at 6 months. 94% avoided bladder removal surgery (cystectomy). 100% were still alive at 6 months.

About Non-Muscle Invasive Bladder Cancer (NMIBC)
NMIBC affects the superficial bladder layer without deeper invasion or metastatic spread. Bladder cancer represents a major clinical challenge as the 13th most commonly diagnosed cancer in Japan and ninth globally,4 with 75% presenting as NMIBC in 2022.4 While intravesical BCG remains first-line standard care for high-risk NMIBC, over 50% of patients experience disease recurrence and progression within one year, with many developing BCG-unresponsive disease requiring radical intervention.5

Current treatment options remain limited for BCG-unresponsive patients, with JUA guidelines recommending only radical cystectomy or clinical trial participation6. Nadofaragene firadenovec offers the first choice non-chemotherapy gene therapy option for these patients, providing an alternative to immediate radical surgery.

On September 11, 2025 Eli Lilly and Company (NYSE: LLY) reported it will participate in the Bernstein’s 2nd Annual Healthcare Forum on September 25, 2025. Lucas Montarce, executive vice president and chief financial officer, will take part in a fireside chat at 8 a.m., Eastern time (Press release, Eli Lilly, SEP 11, 2025, View Source [SID1234655931]).

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A live audio webcast will be available on the "Webcasts & Presentations" section of Lilly’s investor website at View Source A replay of the presentation will be available on this same website for approximately 90 days.