On November 4, 2025 Defence Therapeutics Inc. ("Defence" or the "Company"), a leading biotechnology company specialized in drug delivery technologies, reported at the World ADC Conference in San Diego, USA, highly encouraging results from its latest preclinical in vivo study evaluating Accum-Kadcyla, a novel version of Genentech/Roche’s marketed ADC Kadcyla (ado-trastuzumab emtansine), in mouse models of HER2-positive breast cancer.

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Study Results: 20-Fold Increased Potency at Equivalent Dose

In the comparative in vivo study, Accum-Kadcyla demonstrated a ~20-fold higher anti-tumor efficacy than Kadcyla alone when administered at the same dose (0.5 mg/kg). Tumor growth was significantly halted in the Accum-Kadcyla-treated group, resulting in a durable and near-complete response in most mice while Kadcyla at the same dose (0.5 mg/kg) had no effect on tumor growth. Importantly, 100% of the animals survived throughout the duration of the study with no signs of toxicity, underscoring the excellent tolerability of the treatment.

Implications for Patients and the Industry

These results confirm that Defence’s Accum platform can dramatically enhance the intracellular delivery and potency of ADCs by overcoming endosomal entrapment—a known bottleneck in ADC performance. By increasing the therapeutic payload’s reach inside cancer cells, Accum enables a more efficient drug release and tumor killing, even at lower doses.

This finding is particularly meaningful for patients: the ability to achieve the same or better efficacy at reduced doses translates into a potential reduction in toxicity and side effects, addressing one of the main limitations of current ADC therapies. Practically, it could potentially bring this current second line of treatment to a first line of treatment for the benefit of the patients.

Dr. Maxime Parisotto, Chief Scientific Officer of Defence Therapeutics, commented:

"These results further validate the power of Accum as a transformative technology for ADCs. By amplifying the potency of a clinically proven ADC like Kadcyla by 20 times at the same dose, we demonstrate that Accum can unlock a new generation of safer and more effective targeted therapies for cancer patients."

Next Steps and Commercial Outlook

Following these promising results, Defence Therapeutics plans to expand its Accum-ADC program to additional HER2-positive and HER2-low tumor models and to advance discussions with potential pharmaceutical partners.

(Press release, Defence Therapeutics, NOV 4, 2025, View Source;utm_medium=rss&utm_campaign=defence-therapeutics-demonstrates-strong-preclinical-in-vivo-efficacy-results-evaluating-accum-kadcyla-in-breast-cancer-models [SID1234659633])

On November 4, 2025 Delcath Systems, Inc. (Nasdaq: DCTH) ("Delcath" or the "Company"), an interventional oncology company focused on the treatment of primary and metastatic liver cancers, reported financial results and business highlights for the third quarter ended September 30, 2025.

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Third Quarter 2025 Financial Results

Total revenue of $20.6 million, compared with $11.2 million in the third quarter of 2024
HEPZATO KIT revenue of $19.3 million, compared to $10.0 million in the third quarter of 2024
CHEMOSAT revenue of $1.3 million, compared to $1.2 million in the third quarter of 2024
Gross margins of 87%, compared to 85% in the third quarter of 2024
Net income of $0.8 million, compared to a net income of $1.9 million in the third quarter of 2024
Non-GAAP positive adjusted EBITDA in the third quarter of $5.3 million, compared to a positive adjusted EBITDA of $1.0 million in the third quarter of 2024
Cash provided by operations of $4.8 million in the quarter; compared to $3.6 million used by operations in the third quarter of 2024
Cash and investments of $88.9 million as of September 30, 2025
Business Highlights

There are currently 25 active centers across the U.S.
In August, the first patient was dosed at City of Hope National Medical Center in the global Phase 2 trial of HEPZATO in combination with trifluridine-tipiracil and bevacizumab for liver-dominant metastatic colorectal cancer. The study will enroll approximately 90 patients across 20+ sites in the U.S. and Europe, with topline data expected in 2028
Announced that results from the investigator-initiated Phase 2 CHOPIN trial at Leiden University Medical Center evaluating CHEMOSAT with ipilimumab and nivolumab in metastatic uveal melanoma were presented at the October 2025 European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Annual Congress by Principal Investigator Ellen Kapiteijn, MD, showing a significant improvement in one-year progression-free survival versus CHEMOSAT alone
"In the third quarter, we made strong progress with our clinical programs, reporting compelling positive CHOPIN results and first-patient dosing in our global Phase 2 trial in liver-dominant metastatic colorectal cancer," said Gerard Michel, Chief Executive Officer of Delcath Systems. "While revenue results in the quarter reflected the impact of NDRA discounts and seasonal factors, our fundamentals remain strong. We are confident that the growing clinical validation of HEPZATO positions us well to drive continued progress and long-term value for patients and shareholders."

2025 Full Year Financial Guidance

The Company’s financial outlook for fiscal year 2025:

Total CHEMOSAT and HEPZATO KIT revenue to be in the range of $83 to $85 million, an increase in volume of approximately 150% over 2024
Gross margins in the range of 85% to 87%
Positive adjusted EBITDA and cashflow in each quarter of 2025
Third Quarter 2025 Results

Total revenue for the quarter ending September 30, 2025 was $20.6 million compared to $11.2 million for the same period in the prior year. Revenue in the quarter includes sales of $19.3 million of HEPZATO in the U.S. and $1.3 million of CHEMOSAT in Europe.

Research and development expenses for the quarter ending September 30, 2025, were $8.0 million compared to $3.9 million for the same period in the prior year. The increase is primarily due to costs associated with expanding the clinical team including the share-based compensation expense related to an increase in headcount and initiation of the Phase 2 clinical trial evaluating HEPZATO in combination with standard of care for metastatic colorectal cancer and Phase 2 clinical trial in metastatic breast cancer. In 2024, these costs primarily related to medical affairs and regulatory costs associated with the approved products.

Selling, general and administrative expenses for the quarter ended September 30, 2025, were $10.3 million compared to $7.0 million for the same period in the prior year. The increase is primarily due to continued commercial expansion activities including marketing-related expenses, additional personnel in the commercial team and share-based compensation expenses.

Net income for the quarter ended September 30, 2025 was $0.8 million compared to net income of $1.9 million for the same period in the prior year.

Non-GAAP adjusted EBITDA for the quarter ended September 30, 2025 was $5.3 million compared to adjusted EBITDA of $1.0 million for the same period in the prior year. A table reconciling non-GAAP measures is included in this press release for reference.

As of September 30, 2025, the Company had $88.9 million in cash and investments, and no debt.

Conference Call Information

To participate in this event, dial in approximately 5 to 10 minutes before the beginning of the call.

Event Date: Thursday, November 6, 2025
Time: 8:30 AM Eastern Time

Participant Numbers:
Toll Free: 1-877-407-3982
International: 1-201-493-6780
Webcast: View Source;tp_key=a3bb91787b

A replay of the webinar will be available shortly after the conclusion of the call and will be archived on the company’s website View Source

(Press release, Delcath Systems, NOV 4, 2025, View Source [SID1234659352])

On November 4, 2025 Prelude Therapeutics Incorporated (Nasdaq:PRLD), a clinical-stage precision oncology company, reported an exclusive option agreement with Incyte (Nasdaq:INCY) focused on Prelude’s previously undisclosed mutant selective JAK2V617F JH2 inhibitor program in development for patients with myeloproliferative neoplasms (MPNs). Per the agreement, Incyte secures an exclusive option to acquire the JAK2V617F program in exchange for an upfront payment and a strategic equity investment in Prelude, plus potential downstream milestones and royalties.

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Kris Vaddi, Ph.D., Chief Executive Officer of Prelude stated, "We’re pleased to put this agreement in place with Incyte, recognized global leaders in the MPN field. Prelude and Incyte both aim to deliver transformational treatments to improve upon the standard of care established with first generation JAK2 JH1 inhibitors like Jakafi (ruxolitinib). Our research team made significant progress discovering the first known inhibitors that bind into the JAK2 JH2 ‘deep pocket’ where the V617F mutation resides. These potent and orally bioavailable compounds demonstrate mutant specific inhibition and the potential for disease modification in multiple preclinical models of MPNs. Today’s agreement with Incyte provides us with the capital needed to advance further our JAK2V617F program, while also allowing us to advance the development of our other pipeline programs."

"The agreement with Prelude provides an opportunity to enhance our robust portfolio of clinical and preclinical JAK2V617F candidates for patients with MPNs," said Bill Meury, President and Chief Executive Officer of Incyte. "This transaction aligns with our strategy to develop new and innovative therapies poised to make a meaningful difference for patients."

Terms of the Agreement
Under the terms of the Transaction Agreement, Incyte secures an exclusive option to acquire Prelude’s mutant selective JAK2V617F JH2 inhibitor program, including Prelude’s library of preclinical candidates. Prelude will receive $60 million in capital, comprised of an upfront payment of $35 million, plus a $25 million equity investment by Incyte in Prelude. Incyte will purchase 6.25 million shares of Prelude non-voting common stock at a price of $4.00 per share at deal close. Prelude intends to apply the upfront payment and net proceeds from the sale of the purchased shares to advance the JAK2V617F program and other pipeline assets, and for working capital and general corporate purposes.

Prelude expects to advance the JAK2V617F program to pre-defined milestones. Incyte may elect to exercise its exclusive option during the option period to acquire the program and associated assets from Prelude for $100 million. As the JAK2V617F program candidates advance in the clinic, Prelude would be eligible to receive up to $775 million in additional clinical and regulatory milestones, and single digit royalties on global net sales. Combined, total potential cash payments from the transaction, excluding royalties, could reach up to $910 million.

If Incyte elects to not exercise its option to acquire the program, all JAK2V617F global program rights and interests would remain in the sole ownership and control of Prelude.

Prelude Therapeutics was advised on the transaction by Morgan Lewis & Bockius LLP as legal counsel.

Mutant selective JAK2V617F JH2 inhibitor program
JAK2V617F is the primary driver mutation responsible for disease progression in the majority of patients living with myeloproliferative neoplasms (MPNs). The mutation impacts approximately 95% of patients with polycythemia vera (PV), 60% of patients with essential thrombocythemia (ET) and 55% of patients with myelofibrosis (MF). Identifying JAK2 JH2 inhibitors that selectively target V617F+ cells has long been a shared goal and challenge for industry. If successful, this approach has potential to reduce mutant allele burden, modify disease progression, and transform treatment outcomes for MPN patients. Prelude has discovered novel allosteric inhibitors that bind into the JAK2 JH2 "deep pocket" where the V617F mutation resides. These candidates demonstrate mutant specific inhibition in multiple preclinical models of MPNs. The first disclosure of program data was accepted for oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) 67th Annual Meeting taking place in Orlando, FL December 6-9, 2025. The abstract can be found on the ASH (Free ASH Whitepaper) 2025 website ASH (Free ASH Whitepaper) Annual Meeting & Exposition – Hematology.org.

(Press release, Prelude Therapeutics, NOV 4, 2025, View Source [SID1234659368])

On November 4, 2025 Opna Bio, a clinical-stage biopharmaceutical company focused on the discovery and development of novel oncology therapeutics, reported that it will have an oral and a poster presentation at the upcoming 67th Annual American Society for Hematology (ASH) (Free ASH Whitepaper) conference, taking place December 6-9, 2025, in Orlando, FL. The presentations will focus on the company’s novel, multi-functional protein degrader program and OPN-2853, a bromodomain and extra-terminal motif (BET) inhibitor currently being tested in a Phase 1 combination study with ruxolitinib in patients with advanced myelofibrosis. Presentation details are included below.

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The oral presentation will feature preclinical data from Opna’s protein degrader program, which is focused on creating novel therapeutics designed to block multiple oncogenic targets – EP300, CBP, IKZF1 and IKZF3 – concurrently in the same cancer cell. The data highlights the preclinical compound’s potential for activity as a single agent in hematological malignancies such as multiple myeloma and lymphoma.

"Our protein degrader program builds on compelling preclinical data presented at ASH (Free ASH Whitepaper) in 2024 showing strong synergy when combining immunomodulatory (IMiD) drugs and OPN-6602," said Gideon Bollag, PhD, chief scientific officer of Opna Bio. "OPN-6602, an oral, small molecule EP300/CBP inhibitor, is currently being tested in a Phase 1 study in patients with relapsed or refractory multiple myeloma at multiple sites in the U.S."

The poster presentation will highlight updated interim data from the ongoing Phase 1 study of OPN-2853 in patients with myelofibrosis who are no longer responding to ruxolitinib. This investigator-initiated study is led by Professor Adam Mead at the University of Oxford through a collaboration with Cancer Research UK (CRUK) and is run through the Cancer Research UK Clinical Trials Unit at the University of Birmingham.

ASH Presentation Details:

Title: Novel multifunctional degraders of EP300/CBP and IKZF1/3 with potent anti-myeloma activity
Publication Number: 573
Session Name: 651. Multiple Myeloma and Plasma Cell Dyscrasias: Basic and Translational: Emerging Myeloma Disease Mechanisms and Therapeutic Strategies
Date and Session Time : December 7, 2025, 12:00 PM – 1:30 PM ET
Presentation Time: 12:30 PM – 12:45 PM ET
Presenter: Pan-Yu Chen, PhD, Associate Director, Translational Medicine, Opna Bio

Title: Interim analysis of PROMise, a clinical study combining the BET inhibitor OPN-2853 with ruxolitinib in patients with advanced myelofibrosis experiencing an inadequate response to ruxolitinib
Publication Number: 3794
Session Name: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster II
Date and Session Time: December 7, 2025, 6:00 PM – 8:00 PM ET
Presentation Time: 6:00 PM – 8:00 PM ET
Presenter: Adam Mead, PhD, Professor of Haematology, Radcliffe Department of Medicine, CRUK Senior Cancer Research Fellow

(Press release, Opna Bio, NOV 4, 2025, View Source [SID1234659402])

On November 4, 2025 Relmada Therapeutics, Inc. (Nasdaq: RLMD, "Relmada" or the "Company"), a clinical-stage biotechnology company advancing innovative therapies for oncology and central nervous system indications, reported the receipt of written minutes from a Type B pre-IND meeting with the U.S. Food and Drug Administration (FDA) regarding the planned Phase 3 program for NDV-01 in non-muscle invasive bladder cancer (NMIBC) patients. The Company will be requesting follow-up meetings with FDA to discuss each development path. Relmada secured FDA alignment on certain key elements of the planned Phase 3 pivotal program for NDV-01, expected to begin in H1 2026 and incorporating two independent studies for approval in two separate indications:

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High-grade, 2nd line BCG-unresponsive NMIBC patients
Intermediate risk NMIBC in the adjuvant setting
Key Outcomes from the FDA Type B pre-IND meeting (specific study design details to be further discussed with the agency):

In high-grade, 2nd line BCG-unresponsive setting, the FDA stated that a single arm trial might be acceptable in a more refractory patient population.
In the intermediate risk NMIBC setting, the FDA agreed that a proposal to randomize patients post-TURBT to adjuvant NDV-01 vs observation, evaluating a time-to-event endpoint, is generally acceptable.
Further non-clinical studies are not required. FDA indicated that no further non-clinical studies are required to support a 505(b)(2) New Drug Application (NDA).
"The positive outcome of our Type B meeting and alignment with the FDA on the Phase 3 pivotal program mark a key milestone for Relmada and NDV-01," said Raj Pruthi, MD, Chief Medical Officer – Urology, Relmada Therapeutics. "We believe the FDA’s guidance provides a path to advance NDV-01 for patients with NMIBC who currently have limited options. We believe a single-arm registrational study in high-grade, refractory BCG-unresponsive patients offers a rapid route to potential approval, while alignment on a separate second pivotal study in intermediate-risk NMIBC could enable an additional indication and broader clinical adoption."

Sergio Traversa, Chief Executive Officer of Relmada Therapeutics, stated: "We added NDV-01 to our portfolio based on its strong potential to transform the treatment of NMIBC. The outcome of our Type B meeting with the FDA further reinforces our confidence in the path forward and in NDV-01’s potential to become a best-in-class, durable, ready and easy-to-use, in-office, bladder-sparing therapy. We look forward to initiating the Phase 3 programs in the first half of 2026."

Also, Relmada announced 9-month follow-up data from the Phase 2 study of NDV-01 in non-muscle invasive bladder cancer.

Highlights of the 9-month follow-up data and updated 3-month and 6-month data from the Phase 2 study of NDV-01:

Clinical Results (Response Data)
Complete Response % (n/N)
Anytime 92% (23/25)
3 months 84% (21/25)
6 months 87% (20/23)*
9 months 85% (17/20)*
*Includes patients with CR after re-induction. 60% CR rate after re-induction.

Two subjects have reached 12-month assessment, and both have a CR
No patient had progression to muscle invasive disease
No patient underwent a radical cystectomy
No new safety signals in terms of type, number, or degree of AEs — with no patients having a >= Grade 3 TRAE and no patients discontinued treatment due to AEs
36 enrolled patients (receiving >= 1 dose), of which 22 (61%) experienced a treatment-related AE. Among treatment-related AEs, 62% were transient uncomfortable urination (dysuria), 9% were asymptomatic positive urine culture and 7% were hematuria.
Efficacy in BCG-Unresponsive Subpopulation**:

Clinical Results (Response Data)
Complete Response % (n/N)
Anytime 91% (10/11)
3 months 82% (9/11)
6 months 78% (7/9)
9 months 88% (7/8)
n = 18 patients dosed in BCG-UR subpopulation
BCG-UR defined by FDA definition**
BCG-UR, Bacillus Calmette-Guérin (BCG) – Unresponsive

**View Source

About NDV-01

NDV-01 is a sustained-release, intravesical formulation of gemcitabine and docetaxel (Gem/Doce), in development for the treatment of non-muscle invasive bladder cancer. It is designed to enable Gem/Doce bladder retention and gradual drug release over 10 days. The formulation creates a soft matrix that enhances local tumor exposure. NDV-01 is ready to use, convenient to administer in-office in less than 10 minutes, and does not require preparation, anesthesia or specialized equipment.

About NMIBC

NMIBC represents 75-80% of all bladder cancer cases and is associated with high recurrence (50 –80% over 5 years). With over 744,000 prevalent cases in the U.S. and limited treatment options, the market opportunity is significant. NDV-01 has the potential to serve as a frontline or salvage therapy and could be applicable across multiple NMIBC subtypes.

(Press release, Relmada Therapeutics, NOV 4, 2025, View Source [SID1234661915])