On September 9, 2025 Tubulis reported the appointment of Halley Gilbert, JD, as Chief Legal Officer and Chief Operating Officer (CLO/COO) (Press release, Tubulis, SEP 9, 2025, View Source [SID1234655896]). Bringing over 20 years of transaction and operations experience in the biopharmaceutical industry, Ms. Gilbert will further expand Tubulis’ leadership capabilities at a pivotal stage of clinical and corporate development. In this newly created role, she will leverage her combined expertise as an attorney and seasoned biotech executive to shape Tubulis’ strategy and support the continued growth of the company’s U.S. presence. Ms. Gilbert will be responsible for legal and administrative functions and play a key role in executing strategic transactions for the company.

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"Halley’s extensive legal and operational experience and deep commitment to supporting rapidly growing biotech companies make her an ideal addition to our team as we further expand our clinical and business operations," said Dr. Dominik Schumacher, CEO and Co-founder of Tubulis. "Her industry know-how and strategic leadership will be instrumental as we advance our lead ADC candidates through clinical development and continue to expand our pipeline with new modalities."

"ADCs are transforming the oncology landscape, and Tubulis is leading the way with its differentiated approach to ADC design," said Halley Gilbert, JD, CLO and COO of Tubulis. "Their innovative platforms and exciting research ethos allow them to unlock the full therapeutic potential of this powerful drug class. I am thrilled to be joining such a motivated team and look forward to helping shape the path toward commercialization and delivering a meaningful clinical impact to patients."

Ms. Gilbert brings extensive legal and operational experience within the life sciences industry, having served as CLO and COO at both public and private biotech companies. Most recently, she served as Chief Legal Officer of Cargo Therapeutics and played a key leadership role in the company’s initial public offering and subsequent sale. Prior to Cargo, Ms. Gilbert was the first employee of Invyvid, Inc. (formerly Adagio), where she enabled the company’s rapid growth from launch to IPO, and accelerated its transition from early R&D to late-stage clinical development. In addition, for the past five years, Ms. Gilbert has been a member of the Board of Directors at Vaxcyte, Inc., Arcutis Biotherapeutics, and CytomyX Therapeutics, Inc.

On September 9, 2025 Tempus AI, Inc. (NASDAQ: TEM), a technology company leading the adoption of AI to advance precision medicine, reported the publication of a study in JCO Precision Oncology validating the clinical utility of the company’s PurIST algorithmic diagnostic (Press release, Tempus, SEP 9, 2025, View Source [SID1234655894]). The study provides the largest real-world evidence to date supporting the integration of PurIST into routine clinical care for patients with advanced PDAC, with the aim of informing first-line chemotherapy selection and improving patient outcomes.

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Pancreatic cancer remains one of the most lethal malignancies, with limited therapeutic options and a five-year survival rate of just 12%. For patients with advanced, unresectable PDAC, the two most common first-line chemotherapy regimens, FOLFIRINOX (FFX) and gemcitabine plus nab-paclitaxel (GnP), have shown variable efficacy, and clinicians have lacked robust biomarkers to guide optimal therapy selection. To address this challenge, Tempus collaborated with GeneCentric to develop and deploy PurIST, a clinically validated, RNA-based algorithm test that classifies PDAC tumors as either "classical" or "basal" subtypes.

The Tempus-led study analyzed a real-world cohort of 931 patients with advanced PDAC, using the Tempus xR RNA sequencing platform to assign PurIST subtypes. Patients were treated with either first-line FFX or GnP, and clinical outcomes were assessed according to PurIST classification. The study’s findings establish PurIST as both a prognostic and predictive biomarker, enabling clinicians to personalize first-line therapy for advanced PDAC patients and maximize the likelihood of improved survival.

Prognostic Value: Among patients treated with FFX (N=536), those with the classical subtype had a significantly longer median overall survival (OS) of 11.8 months, compared to 7.0 months for basal subtype patients (Hazard Ratio [HR]=1.86; p<0.001).
Predictive Value: In patients with the classical subtype and good performance status (ECOG 0 or 1, N=311), treatment with FFX was associated with a 33% relative risk reduction in death compared to GnP (HR=0.67; p<0.009). No comparable benefit was observed in basal subtype patients.
"Pancreatic cancer is a challenging disease, and the ability to match patients to the most effective first-line therapy is critical. Prior research has suggested that certain molecular subtypes are associated with distinct prognoses and responses to therapy, but until now, large-scale real-world validation in a clinical setting has been lacking," said Ezra Cohen, MD, Chief Medical Officer, Oncology. "Our study demonstrates that PurIST subtyping enables a biomarker-driven approach to therapy selection, addressing a major unmet need in the management of pancreatic cancer."

"PurIST is a step forward for pancreatic cancer molecular testing, giving healthcare providers a new tool to better inform personalized treatment options for this devastating disease," said Michael Milburn, PhD, President and CEO of GeneCentric and co-author on the publication. "GeneCentric’s collaboration with Tempus allowed us to validate the algorithm leveraging the power of Tempus’ multimodal data to rapidly deliver a much-needed test for patients with PDAC."

On September 9, 2025 DeuterOncology, a clinical-stage biotech company focused on developing best-in-class deuterated cancer therapies, reported compelling safety, pharmacokinetic and efficacy results from its ongoing Phase I dose escalation study of DO-2, a novel deuterated MET tyrosine kinase inhibitor (Press release, DeuterOncology, SEP 9, 2025, View Source [SID1234655892]). The results are being presented at the World Conference on Lung Cancer (WCLC) 2025 in Barcelona (Abstract ID-2859).

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The Phase I study enrolled 29 patients (26 RECIST 1.1 evaluable) with advanced solid tumors harboring any known driver alterations, with 86% having non-small cell lung cancer (NSCLC). The study’s primary findings demonstrate that DO-2 treated patients showed a remarkable 100% Disease Control Rate (DCR) and saw tumor shrinkage in 100% of MET exon 14 skip mutation-positive NSCLC patients without alternative oncogenic drivers and reached efficacious drug exposures (10/10 patients). RECIST 1.1 Partial responses (>30% reduction) were observed in 2 out of these 10 patients. Importantly, the study showed a markedly improved safety profile compared to approved MET inhibitors, with only one patient (5%) experiencing grade 1 peripheral oedema versus 68-82% seen with other agents in this class.

"These results represent a significant advancement in MET-targeted therapy," said Dr. Cecilia Ahlin, CMO of DeuterOncology. "Our deuteration strategy has successfully enhanced drug exposure while maintaining efficacy, but most importantly, we have achieved this without the debilitating peripheral oedema that forces many patients to discontinue current MET inhibitors —symptoms that can severely limit daily activities such as walking, dressing, or cooking. This addresses a critical unmet medical need for cancer patients."

The study’s key findings include:

Superior Safety Profile: Only 1 patient (5%) experienced peripheral oedema (grade 1) compared to 68-82% with approved competitors. No grade 3 or 4 peripheral oedema was observed versus ~17% in approved competitors
Enhanced Pharmacokinetics: Deuteration increased plasma exposure compared to the non-deuterated parent compound, enabling once-daily dosing at 60mg with food, versus 450-800mg daily with approved competitors
Promising Efficacy: A 100% disease control rate (DCR) was observed, including two partial responses, in treatment-naïve and previously treated MET exon 14 skipping NSCLC patients without known resistance mechanisms who achieved efficacious drug exposure. Notably, one patient with pleural effusion experienced full resolution and maintained stable disease for over 20 months
Excellent Tolerability: Only two grade 3 treatment-related adverse events (fatigue and reversible creatinine increase) were observed across all dose levels
Dr. Hans Prenen, Principal Investigator at University Hospital Antwerp (UZA), commented: "The clinical data for DO-2 are very encouraging. The fact that we’re seeing robust anti-tumor activity with minimal peripheral oedema represents a potential game-changer for MET-driven lung cancer patients, who currently face difficult treatment tolerability issues with existing therapies."

The study confirms DeuterOncology’s hypothesis that continuous 24/7 MET inhibition is not required for efficacy but is responsible for the class-effect toxicity of peripheral oedema. DO-2’s unique biochemical and pharmacokinetic profile allows for effective target engagement while providing recovery periods that minimize on-target toxicities.

Based on these promising Phase I results, DeuterOncology has initiated an expansion cohort in selected first-line MET exon 14 skip NSCLC patients to further validate the safety and efficacy of the 60mg once-daily regimen.

Details of the poster presentation are as follows:

Title: Preliminary Safety and PK of the MET-TKI DO-2 in Advanced Solid Tumors with MET Aberrations: Phase I Study

Authors: Hans Prenen MD, PhD, Rachel Galot MD, PhD, Alexis Cortot MD, PhD, Aurélie Swalduz MD, PhD, Bernd Dekeyser MD, Peter de Bruijn PhD, Francois Zammit MD, Jean-Pascal Machiels MD, PhD, Brant Delafontaine MD, Sylvie Rottey MD, PhD, Ingrid Desar MD, PhD, Marthe Paats MD, PhD, Barend J. Sikkema3MD, , Florence Wastelin MSc, Cecilia Ahlin MD PhD, Timothy Perera PhD, Jaap Verweij MD, PhD, Carla van Herpen MD, PhD, Debbie G.J. Robbrecht MD, PhD

Presenter: Timothy Perera

Session date and time: 09 September 2025. 10.00AM CEST

Poster ID number: Abstract ID-2859. P3.18.66

About MET-Driven Lung Cancer

Lung cancer remains the most common form of cancer worldwide, affecting approximately 2.2 million individuals annually. MET exon 14 skipping mutations occur in ~3% of NSCLC patients—equating to an estimated 60,000–70,000 new cases worldwide annually—and represent a validated therapeutic target. However, current approved MET inhibitors suffer from high rates of peripheral oedema (>50% of patients), leading to frequent dose reductions, treatment interruptions, and discontinuations that compromise patient outcomes.

On September 9, 2025 ARTBIO, Inc. ("ARTBIO"), a clinical-stage radiopharmaceutical company developing a new class of Pb212 alpha radioligand therapies (ARTs), reported that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for AB001, and confirmed the study may proceed (Press release, ARTBIO, SEP 9, 2025, View Source [SID1234655891]). The news follows a recent Series B capital raise to advance the company’s pipeline and proprietary technology platform for Pb212-based therapies.

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"AB001 is a novel PSMA-targeted and Pb212-based radioligand therapy designed to treat patients with metastatic prostate cancer, one that we believe has the potential to increase the radioligand dose rate to tumors without higher toxicity to normal tissues," said Margaret Yu, M.D., Chief Medical Officer of ARTBIO. "Following FDA review, our Phase 1 study may now move ahead. This is welcome news for patients who are eagerly awaiting new targeted treatment options and for investigators who are ready to begin screening patients for this trial."

AB001 had been previously administered to patients in a Phase 0 study conducted in Norway with encouraging biodistribution results in those participants. The report on this study was included as part of the IND.

"Despite the availability of many treatment options, metastatic prostate cancer is still not a curable disease, requiring additional innovative treatments," said Oliver Sartor, M.D., Director of the Transformational Prostate Cancer Research Center at East Jefferson General Hospital Cancer Center and ARTBIO Scientific Advisory Board Member. "I’m grateful for ARTBIO’s dedication and passion in this area and am excited about the difference that AB001 could make for patients."

ARTBIO plans to initiate the Phase 1 clinical trial for AB001 across multiple sites in the U.S. by leveraging its distributed manufacturing network of production hubs, including leading radiopharmaceutical CDMOs like SpectronRx, PharmaLogic, and Nucleus Radiopharma.

Clinical trial supply will be enabled by ARTBIO’s proprietary Pb212 generator technology, AlphaDirect, with precursor isotopes being sourced from the U.S. Department of Energy. The company plans to enroll patients outside of the U.S. once additional regulatory approvals are secured.

"This IND clearance represents critical progress in translating the unique biology of Pb212 into patient care," said Dr. Michael Morris, Professor of Medicine, Prostate Section Head at Memorial Sloan Kettering Cancer Center and ARTBIO Clinical Advisory Board Member. "With the ability to target radiation at the cellular level, this program has the potential to establish a new standard of care in metastatic prostate cancer."

About AB001
AB001 is an Alpha Radioligand Therapy (ART) consisting of a prostate-specific membrane antigen (PSMA)-targeted small molecule radiolabeled with Pb212. PSMA is commonly overexpressed in mCRPC and has become an attractive target for imaging agents and therapies.

On September 9, 2025 XtalPi Inc. (Stock Code: 2228.HK) reported that PEP08, a next-generation PRMT5 inhibitor candidate developed through its AI drug discovery collaboration with PharmaEngine, Inc. (TWO:4162), has received regulatory clearances to initiate Phase 1 clinical trials in solid tumors (Press release, XtalPi, SEP 9, 2025, View Source [SID1234655890]). Approvals include Australia’s Human Research Ethics Committee (HREC), recognized by the Australian Therapeutic Goods Administration (TGA), and the food and drug authority of the Taiwan region (TFDA). This milestone triggered a payment to XtalPi under the partnership agreement.

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PRMT5 (Protein Arginine Methyltransferase 5) is a validated synthetic lethality target in tumors harboring homozygous MTAP deletion, which is related to poor prognosis and present in 10-15% of human cancers, including small cell lung cancer (NSCLC), mesothelioma, pancreatic cancer, glioblastoma multiforme (GBM), head and neck cancer, esophageal cancer, and bladder cancer. While PRMT5 inhibition offers a potent mechanism for targeted therapy, first-generation inhibitors face significant challenges in selectivity, resulting in dose-limiting toxicities and unmet medical needs.

Leveraging its integrated drug discovery platform that combines quantum physics, AI, and large-scale robotic experiments, XtalPi partnered with PharmaEngine’s scientific team to conduct de novo drug design. The AI platform generated a multi-million compound library for PRMT5, identifying novel lead series with exceptional potency and selectivity. Following iterative optimization through quantum physics and AI-powered ADMET screening, PEP08 emerged as the preclinical candidate (PCC) and advanced through IND-enabling studies by PharmaEngine.

As a second-generation PRMT5 inhibitor with a novel scaffold, PEP08 demonstrates superior activity and selectivity. Its MTA-cooperative binding mode stabilizes a ternary complex with PRMT5, enabling highly selective target inhibition. This mechanism drives potent synthetic lethality efficacy against MTAP-deleted tumor cells while minimizing off-target effects on normal cells, highlighting significant therapeutic potential.

Preclinical studies revealed PEP08’s markedly improved safety profile compared to first-generation PRMT5 inhibitors, alongside favorable blood-brain barrier penetration and overall developability characteristics. The compound achieved robust in vivo efficacy across multiple animal models. Compared to other clinical-stage second-generation candidates, PEP08 exhibits potential best-in-class properties and compelling combination therapy opportunities. PharmaEngine presented these findings at the 2025 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.

PEP08’s successful regulatory clearance and milestone achievement underscore XtalPi’s platform-driven innovation capabilities and mark a pivotal advancement in its partnership with PharmaEngine. XtalPi remains committed to empowering partners to discover novel drug candidates addressing complex targets, accelerate the development of competitive pipeline projects, and deliver transformative medicines to patients worldwide.