Vertex to Participate in the UBS Global Healthcare Conference on November 11

On October 27, 2025 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported management participation in the UBS Global Healthcare Conference. Reshma Kewalramani, President and Chief Executive Officer will participate in a fireside chat on Tuesday, November 11, 2025, at 10:15 a.m. ET.

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A live webcast of management’s remarks will be available through the Vertex website, www.vrtx.com, in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be archived on the company’s website.

(Press release, Vertex Pharmaceuticals, OCT 27, 2025, View Source [SID1234657039])

Actinium Announces Superior Anti-Tumor Activity of ATNM-400 in Lung Cancer Compared to the Leading First, Second and Third-Line Approved EGFR Mutant Therapies and Mechanistic Synergy with Osimertinib at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics

On October 27, 2025 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of differentiated targeted radiotherapies, reported the presentation of the first ever preclinical data of ATNM-400 in non-small cell lung cancer (NSCLC). ATNM-400 a novel, multi-indication first-in-class antibody radioconjugate armed with the potent alpha-emitter Actinium-225 (Ac-225) is Actinium’s lead solid tumor program, which is also being studied in prostate cancer. The data presented at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) studied ATNM-400 in Epithelial Growth Factor Receptor (EGFR)-mutant NSCLC models.

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ATNM-400 demonstrated superior efficacy with 3-5x greater tumor growth inhibition compared to standard-of-care therapies across EGFR-mutant NSCLC including:

Frontline: Osimertinib (TARGRISSO, AstraZeneca) an EGFR tyrosine kinase inhibitor (TKI)

Second line: Dato-DXd (DATROWAY, AstraZeneca/Daiichi Sankyo) a Trop-2 antibody drug conjugate (ADC)

Third line: Amivantamab (RYBREVANT, J&J), an EGFR-cMET bispecific antibody

ATNM-400 also demonstrated synergistic activity in combination with osimertinib with complete tumor regression in 100% of tumor-bearing animals. The synergistic mechanism of this combination is supported by increased expression of the ATNM-400 target antigen after EGFR inhibition with osimertinib. In addition, previously published data of external beam radiotherapy (EBRT) combined with osimertinib resulted in improved progression free survival (PFS) compared to osimertinib alone, providing a strong clinical rationale for combining targeted alpha-therapy via ATNM-400 with EGFR targeting therapies Sampath et al. (AstraZeneca & UT Southwestern Harold C. Simmons Comprehensive Cancer Center)1.

Dr. Sandip Patel, Professor of Medicine at the University of California San Diego, an author on the poster, stated, "Targeted radiotherapy has transformed the prostate cancer treatment landscape and exemplifies what can be achieved with this technology. ATNM-400 represents a novel and differentiated development candidate for non-small cell lung cancer with EGFR mutations. The positive clinical results with EBRT and osimertinib provide strong support for combining targeted alpha-therapy via ATNM-400 and EGFR therapies to effectively deliver radiation to the target tumors and leverage mechanistic synergies while minimizing off-target effects. I am highly encouraged by these data that demonstrate the potential of ATNM-400 which would be a first-in-class radiotherapy."

Notable ATNM-400 NSCLC Data

ATNM-400 produced superior tumor growth inhibition (TGI) in animals bearing human lung cancer NCI-H1975, which carry L858R and T790M EGFR mutations

TGI with ATNM-400 was 3-5 times higher than what was achieved with the current standard of care EGFR-mutant NSCLC therapies including TAGRISSO, DATROWAY and RYBREVANT

The combination of ATNM-400 and osimertinib produced cures in all tumor-bearing animals with complete tumor regression, outperforming either agent alone and suggesting synergistic activity

The ATNM-400 target antigen is overexpressed in NSCLC and is linked to treatment resistance including to osimertinib

Osimertinib treatment significantly increased the expression of the ATNM-400 target antigen in NCI-H1975 lung cancer cells

This increased target expression post-osimertinib notably increased ATNM-400’s in vitro cytotoxic effect when combined post-osimertinib
Sandesh Seth, Actinium’s Chairman and CEO, stated, "Improving outcomes for patients with non-small cell lung cancer remains a major challenge evidenced by the significant number of therapies and modalities in development. ATNM-400 represents a truly innovative approach that leverages the potent Ac-225 alpha-emitter payload against a target that is overexpressed in lung cancer and linked to treatment resistance. These results strongly support ATNM-400’s differentiated profile and its potential for development in first, second and third-line treatment settings, alone or in combination with some of the most successful drugs that compete with each other in the EGFR-mutant segment. ATNM-400’s potential against difficult to treat and common mutations in lung cancer and the recent data updates in prostate cancer at the Prostate Cancer Foundation meeting further validate the multi-tumor potential of ATNM-400 in several disease and treatment settings that support several blockbuster drugs. We look forward to providing further updates on this exciting pipeline candidate as we progress into clinical development."

Potential for ATNM-400 in NSCLC

Lung cancer is the most common cancer worldwide with more than 200,000 new cases estimated in 2025 in the U.S. and over 2 million new cases globally. NSCLC accounts for approximately 85 percent of all lung cancer cases with EGFR mutations detected in approximately 10-15 percent of Western cases and 40-60 percent of some Asian populations. Approved therapies for EGFR-mutant NSCLC collectively generated approximately $7 billion in sales in 2024, including EGFR tyrosine kinase inhibitor osimertinib (TARGRISSO, AstraZeneca) in the frontline setting that led the class with sales of $6.6 billion in 2024, Trop-2 antibody drug conjugate Dato-DXd (DATROWAY, AstraZeneca/Daiichi Sankyo) that was approved in January 2025 in the second line setting, and the EGFR-cMET bispecific antibody amivantamab (RYBREVANT, J&J) in later lines that had sales of $0.3 billion in 2024. Despite their success, nearly all patients eventually develop resistance within two to three years, underscoring the urgent need for novel, differentiated therapies to overcome resistance and improve survival outcomes. ATNM-400 is designed to address this significant unmet need by combining the high-affinity targeting of a monoclonal antibody directed against an antigen overexpressed in NSCLC, associated with poor prognosis and osimertinib resistance, with the potent alpha-emitting isotope Ac-225, which induces irreversible double-strand DNA breaks. The preclinical data showed ATNM-400’s differentiated potential as monotherapy, combination therapy with EGFR-inhibitors like osimertinib and in post-EGFR-resistance settings.

About ATNM-400

ATNM-400 is a highly innovative, first-in-class, and multi-indication Actinium-225 (Ac-225) targeted radiotherapy candidate in development for prostate cancer and non-small cell lung cancer (NSCLC). ATNM-400 is highly differentiated in prostate cancer as it targets a distinct non-PSMA protein strongly implicated in prostate cancer disease biology including progression and treatment resistance. Unlike 177Lu-PSMA-617, the active agent in Pluvicto and the majority of radiotherapies under development, which rely on PSMA targeting, ATNM-400 is designed to maintain efficacy in low-PSMA or high-PSMA resistant disease, a major unmet clinical need as up to 30% of patients do not respond to PSMA radioligand therapies and up to 60% of patients have at least one PSMA-negative tumor lesion. Ac-225 delivers high-linear-energy-transfer alpha particles that induce irreparable double-strand DNA breaks, offering superior potency over beta emitters like Lutetium-177 (177Lu), and has a shorter tissue path length that may reduce off-target toxicity. The receptor specifically targeted by ATNM-400 continues to be expressed at a high level even after androgen receptor inhibitor (ARPI) and ATNM-400 has shown to overcome resistance to the ARPI therapy enzalutamide and work synergistically in combination with enhanced tumor control including complete tumor regression. In NSCLC, ATNM-400 has shown superior efficacy to approved first, second and third line EGFR therapies including small molecules, antibody drug conjugates and bispecific antibodies that is synergistic with osimertinib, an EGFR tyrosine kinase inhibitor (TKI) that is a standard of care therapy approved for treatment of patients in the frontline setting, and is also able to overcome osimertinib resistance.

Prostate cancer is the most commonly diagnosed cancer in men, with ~1.5 million new cases globally and over 313,000 expected in the U.S. in 2025. While early-stage disease is typically managed with surgery, radiation, and ARPI therapy, up to 20% of cases progress to mCRPC – a lethal stage with limited treatment options. Targeted radiotherapy is a growing field in prostate cancer, dominated by PSMA-targeting agents like Pluvicto, which had sales of over $1.3 billion in 2024, yet many patients either lack PSMA expression or develop resistance to Pluvicto. In the U.S., 40,000–60,000 mCRPC patients annually progress after ARPI therapy, which as a class had sales of over $10.0 billion in 2024 including enzalutamide (Xtandi) that led the ARPI class with sales of over $5.9 billion in 2024, highlighting a significant unmet need. Lung cancer is the leading cause of cancer deaths and there are there are over 200,000 new cases expected in the U.S. in 2025 and over 2 million cases globally. NSCLC accounts for approximately 85% of all lung cancer cases. EGFR targeting therapies had sales of approximately $7 billion in 2024 with the EGFR TKI Osimertinib (TAGRISSO, AstraZeneca) generating sales of $6.6 billion in 2024. Across prostate cancer and NSCLC, there are approximately 500,000 new cases in the U.S. alone.

(Press release, Actinium Pharmaceuticals, OCT 27, 2025, View Source [SID1234657038])

Revolution Medicines’ RAS(ON) Multi-Selective Inhibitor Daraxonrasib Granted U.S. FDA Orphan Drug Designation in Pancreatic Cancer

On October 27, 2025 Revolution Medicines, Inc. (Nasdaq: RVMD), a late-stage clinical oncology company developing targeted therapies for patients with RAS-addicted cancers, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to daraxonrasib, the company’s RAS(ON) multi-selective inhibitor, for the treatment of pancreatic cancer.

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"We are gratified the FDA has granted Orphan Drug Designation to daraxonrasib for the treatment of pancreatic cancer, a devastating disease with limited therapeutic options and representing a large unmet medical need," said Mark A. Goldsmith, M.D., Ph.D., chief executive officer and chairman of Revolution Medicines. "RAS driver mutations are present in nearly all pancreatic cancer cases, underscoring the urgent need for innovative therapies that target this critical driver of disease progression."

Daraxonrasib is being studied in a global Phase 3 clinical trial, RASolute 302, in patients with second line metastatic pancreatic ductal adenocarcinoma (PDAC). The company has also announced plans to initiate two additional Phase 3 clinical trials in pancreatic cancer: a trial for first line treatment in patients with metastatic PDAC and a trial for adjuvant treatment in patients with resectable PDAC.

Orphan Drug Designation is awarded by the FDA on behalf of selected investigational drugs to encourage the development of therapies for rare diseases, which are defined as conditions affecting fewer than 200,000 individuals in the United States. This designation provides sponsors with several incentives, including tax credits for clinical trial costs, exemption from certain FDA fees, and up to seven years of market exclusivity following approval.

About Pancreatic Cancer and Pancreatic Ductal Adenocarcinoma
Pancreatic cancer is one of the most lethal malignancies, characterized by its typically late-stage diagnosis, resistance to standard chemotherapy, and high mortality rate. In the U.S., recent estimates indicate that approximately 60,000 people will be diagnosed annually with pancreatic cancer1, and about 50,000 people will die from this aggressive disease.

Due to the lack of early symptoms and detection methods, approximately 80% of patients are diagnosed with PDAC at an advanced or metastatic stage. It is the most commonly RAS-addicted of all major cancers, and more than 90% of patients have tumors that harbor RAS mutations2. Metastatic PDAC remains one of the most common causes of cancer-related deaths in the U.S., with a five-year survival rate of approximately 3%3.

About Daraxonrasib
Daraxonrasib (RMC-6236) is an oral, direct RAS(ON) multi-selective inhibitor with the potential to help address a wide range of cancers driven by oncogenic RAS mutations. Daraxonrasib suppresses RAS signaling by blocking the interaction of RAS(ON) with its downstream effectors. It does so by targeting oncogenic RAS mutations G12X, G13X and Q61X that are common drivers of major cancers, including pancreatic ductal adenocarcinoma (PDAC), non-small cell lung cancer (NSCLC) and colorectal cancer (CRC).

(Press release, Revolution Medicines, OCT 27, 2025, View Source [SID1234657037])

Rakovina Therapeutics Showcases Potent AI-Developed ATR Inhibitors with CNS Penetrance at AACR-NCI-EORTC International Conference

On October 27, 2025 Rakovina Therapeutics Inc. ("Rakovina" or the "Company") (TSX-V: RKV)(FSE: 7JO0) reported the successful presentation of new data at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper), held October 23–27 in Boston, Massachusetts. The Company’s poster, titled "Novel ATR inhibitors with CNS penetrance developed by artificial intelligence," highlights promising results from its AI-driven kt-5000AI drug discovery program for Ataxia telangiectasia and Rad3-related protein serine/threonine kinase (ATR) inhibitors for cancer therapy.

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Through its collaboration with Variational AI, Rakovina Therapeutics utilized the Enki generative AI platform to identify novel small-molecule candidates predicted to meet a defined target product profile for CNS-penetrant ATR inhibition. Data presented at the conference confirm that multiple lead compounds achieved this profile, demonstrating potent ATR inhibition together with clear evidence of central nervous system exposure.

While multiple ATR inhibitors are in development globally, none have yet demonstrated meaningful central nervous system penetration. Rakovina’s kt-5000AI program is advancing next-generation, AI-designed ATR inhibitors with confirmed CNS exposure, potentially extending the benefits of ATR-targeted therapy to patients with primary brain tumors and brain metastases, where effective treatments remain limited.

In cell-based assays, multiple compounds demonstrated:

>50% inhibition of ATR activity below 200 nM;
potency exceeding reference compounds such as ceralasertib, tuvusertib, and elimusertib;
equal selectivity across the PIKK kinase family compared to reference compounds; and
metabolic stability following incubation with human liver microsomes
In in vivo pharmacokinetic studies, lead compounds demonstrated:

measurable drug concentrations in both plasma and brain tissue following intraperitoneal dosing at 5 mg/kg, indicating CNS penetration; and
good tolerability after single-dose administration, supporting continued optimization and preclinical development
"Presenting at AACR (Free AACR Whitepaper)-NCI-EORTC is an opportunity to highlight Rakovina’s progress in translating AI-discovered compounds into preclinical validation to the scientific community," said Prof. Mads Daugaard, President and Chief Scientific Officer of Rakovina Therapeutics. "ATR is a central regulator of DNA damage repair and a validated target across multiple tumor types. Developing ATR inhibitors capable of penetrating the blood–brain-barrier could expand treatment options for patients with primary brain tumors and brain metastases, areas where few effective therapies exist."

(Press release, Rakovina Therapeutics, OCT 27, 2025, View Source;utm_medium=rss&utm_campaign=rakovina-therapeutics-showcases-potent-ai-developed-atr-inhibitors-with-cns-penetrance-at-aacr-nci-eortc-international-conference [SID1234657036])

ORIC® Pharmaceuticals Presented Preclinical Data at the EORTC-NCI-AACR International Conference on Molecular Targets and Cancer Therapeutics Supporting Best-in-Class Potential of ORIC-944 to Treat Patients With Prostate Cancer and Other Solid Tumors

On October 27, 2025 ORIC Pharmaceuticals, Inc. (Nasdaq: ORIC), a clinical stage oncology company focused on developing treatments that address mechanisms of therapeutic resistance, reported posters at the 2025 EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) highlighting preclinical data that further illustrate the potential for ORIC-944, a potent and selective allosteric inhibitor of the polycomb repressive complex 2 (PRC2) via the embryonic ectoderm development (EED) subunit, to treat prostate cancer and various other solid tumors.

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"These preclinical data underscore the potential of ORIC-944 to overcome resistance not only in prostate cancer but in other solid tumors and highlight that the therapeutic potential of PRC2 inhibition may be maximized in combination with inhibitors of key tumor drivers, including AR inhibitors and KRAS inhibitors," said Lori Friedman, PhD, chief scientific officer. "Based on these data and clinical findings to date, we continue to believe ORIC-944 is a potential best-in-class PRC2 inhibitor with potential in both castration-resistant and castration-sensitive prostate cancer, as well as multiple other tumor types."

Key findings of posters presented at the 2025 EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper):

PRC2 inhibition enhances AR inhibitor response to delay treatment relapse in castration-sensitive prostate cancer by restricting adaptation of tumor cells in preclinical studies

ORIC-944 is a potential best-in-class PRC2 inhibitor that, when combined with androgen receptor (AR) inhibition, synergistically impaired tumor growth, significantly improved survival and extended the duration of response to AR inhibitors in vivo by restricting cellular plasticity and delaying prostate tumor adaptation in castration-sensitive prostate cancer (CSPC).
Transcriptional effects induced by combining ORIC-944 and AR inhibitors were comparable across all AR inhibitors tested (i.e., darolutamide, apalutamide or enzalutamide), and consistent with transcriptional effects of mevrometostat and AR inhibitor combination.
Mechanistically, ORIC-944 in combination with AR inhibition in CSPC was linked to increased luminal cell state, and the restriction of lineage adaptation through reduced chromatin accessibility at binding sites of transcription factors associated with lineage diversification and cell plasticity such as FOXA, HNF1A and ONECUT2. These results were reproduced with mevrometostat and AR inhibitor combination and are consistent with what was previously reported in preclinical studies of CRPC.
PRC2 inhibition enhances KRAS inhibitor response to delay treatment relapse in KRAS-mutant preclinical lung and colorectal cancer models

ORIC-944 is a potential best-in-class PRC2 inhibitor that, when combined with KRAS inhibition, significantly improved efficacy and progression-free survival in KRAS G12C mutant non-small cell lung cancer (NSCLC) and colorectal cancer (CRC) models, demonstrating that PRC2 inhibition can deepen and extend responses by preventing or delaying resistance to KRAS inhibition.
PRC2 activity is increased in tumors from KRAS-mutant NSCLC and CRC patients, and transcriptional analysis from in vivo studies of CRC demonstrated that PRC2 inhibition drives tumor cell differentiation.
ORIC-944 combined with the KRAS inhibitor adagrasib, regressed 100% of tumors in KRAS-mutant adenocarcinoma and squamous NSCLC xenograft models in vivo. The combination also prevented adagrasib tumor relapse and extended progression-free survival in a KRAS G12C adenocarcinoma NSCLC xenograft model.

About ORIC-944
ORIC-944 is a potent and selective allosteric PRC2 inhibitor via EED subunit that demonstrates best-in-class drug properties in preclinical studies, including potency, solubility, and pharmacokinetics, with half-life supporting once daily dosing. ORIC-944 was initially evaluated as a single agent in a Phase 1b trial in patients with advanced prostate cancer and demonstrated potential best-in-class drug properties, including clinical half-life of approximately 20 hours, robust target engagement, and a favorable safety profile. ORIC-944 continues to further demonstrate a potential best-in-class profile with positive interim PSA response data generated in an ongoing Phase 1b trial in combination with ERLEADA (apalutamide) and in combination with NUBEQA (darolutamide) for prostate cancer (NCT05413421).

(Press release, ORIC Pharmaceuticals, OCT 27, 2025, View Source [SID1234657035])