1stOncology™: Cancer Intelligence Service – Page 150 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Pasithea Therapeutics Presents Updated Interim Data from Ongoing Phase 1 Study of PAS-004 at the ASCO Annual Meeting 2025

On June 2, 2025 Pasithea Therapeutics Corp. (NASDAQ: KTTA) ("Pasithea" or the "Company"), a clinical-stage biotechnology company developing PAS-004, a next-generation macrocyclic MEK inhibitor, reported updated interim results from its ongoing dose escalation Phase 1 study evaluating PAS-004 in advanced cancer patients in a poster presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2025 Annual Meeting (Press release, Pasithea Therapeutics, JUN 2, 2025, View Source [SID1234653619]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

Schedule Your 30 min Free Demo!

The Phase 1 clinical trial is a multi-center, open-label, dose escalation, modified 3+3 study design to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and preliminary efficacy of PAS-004 in patients with MAPK pathway-driven advanced solid tumors with a documented RAS, NF1 or RAF mutation, or patients who have failed BRAF/MEK inhibition (NCT06299839).

As of the cut-off date of April 2, 2025, a total of 21 patients had been enrolled and received at least one dose of PAS-004 in six cohorts (Capsules: 2mg, 4mg, 8mg, 15mg, 22mg / Tablets: 4mg). The most common cancer diagnosis was pancreatic cancer (28.6%), colorectal cancer (28.6%), and melanoma (23.8%).

All treatment-related adverse events (AEs) have been either grade 1 or grade 2. No known MEK inhibitor class-related AEs such as ocular toxicities, cardiotoxicities, and skin toxicities were observed during the DLT observation period. No DLTs were reported, and dose escalation is ongoing.

Preliminary PAS-004 PK analysis suggests linear PK with an estimated half-life in excess of 60 hours. The Cmax (peak) to Cmin (trough) ratio was below 2 at steady state in all dose levels and has achieved potentially sufficient exposures for target engagement. This is supported by previously reported preliminary pERK inhibition observed in cohort 3 (8mg capsule), with pERK inhibition of up to 91%.

PAS-004 has demonstrated a dose-dependent PK profile and preliminary clinical activity as a monotherapy in patients with heavily pre-treated, refractory solid tumors. In the efficacy evaluable population (n=16), early response evaluation reveals stable disease (SD) by RECIST 1.1 in 10 patients at some point during the trial, with progression free survival of up to 159 days and overall survival of up to 253 days. In Cohort 4A (15mg capsule), two out of three patients achieved stable disease and remain on therapy. One patient with stage 4 KRAS G12R-mutated pancreatic cancer, having progressive disease while on three prior lines of therapy, achieved a tumor diameter reduction of -9.8% and remains on study for over 5 months. The second patient with Stage 4 BRAF-mutated melanoma, having progressed on two prior lines of therapy, including a prior MEK inhibitor + BRAF inhibitor combination treatment, achieved tumor diameter reduction of -14.9% and remains on study for over 5 months.

"The interim results from our ongoing Phase 1 study are encouraging and we believe underscore the potential of PAS-004 as a best-in-class MEK inhibitor to serve patients with a broad range of MAPK pathway driven tumors," said Dr. Tiago Reis Marques, Chief Executive Officer of Pasithea. "MEK inhibitors have been a transformative class of treatment therapies and we continue to witness groundbreaking advances and new approvals with this class of drug across tumor types and mutational profiles. At ASCO (Free ASCO Whitepaper) 2025, over 15 data sets featuring MEK inhibitors are being presented underscoring the growing momentum in this field. Additionally, we have recently seen approval of two novel MEK inhibitors, mirdametinib and avutometinib, highlighting the continued relevance of this drug class in the past several months alone. As a macrocyclic compound, PAS-004 potentially represents a significant advancement in the MEK inhibitor field by offering high selectivity and sustained pathway suppression while maintaining good tolerability. This profile may make it optimal for both monotherapy and combination therapy, including in patients who have failed prior MEK inhibitors."

The poster presentation will be available on the Pasithea website on the date of the poster session.

Sanofi Exercises License Extension Option to Nurix’s STAT6 Program

On June 2, 2025 Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of targeted protein degradation medicines, reported that Sanofi has exercised its option to exclusively license Nurix’s STAT6 program, including the drug development candidate NX-3911, an oral, highly selective STAT6 degrader (Press release, Nurix Therapeutics, JUN 2, 2025, View Source [SID1234653618]). STAT6 (signal transducer and activator of transcription 6) plays a central role in type 2 inflammation, which drives diseases such as atopic dermatitis and asthma.

"Using our DEL-AI platform, we identified novel DEL-derived chemical matter from which we developed, together with Sanofi, a potential best-in-class STAT6 degrader, NX-3911, which achieves rapid and complete STAT6 degradation," said Gwenn M. Hansen, Ph.D., chief scientific officer of Nurix. "NX-3911 is a potent, selective, orally administered degrader of STAT6 that shows robust efficacy in multiple preclinical models of atopic dermatitis and asthma, demonstrating anti-inflammatory efficacy in animal models equivalent to a STAT6 gene knockout."

"This is the second license extension of a Nurix autoimmune disease program by Sanofi in the last 90 days, highlighting the power of our proprietary DEL-AI drug discovery platform to fuel the discovery of novel medicines to a range of therapeutically important targets like STAT6," said Arthur T. Sands, M.D., Ph.D., president and chief executive officer of Nurix. "Notably, our STAT6 program also includes additional differentiated discovery-stage assets, which could represent an additional product opportunity within our Sanofi collaboration."

Under the 2019 collaboration agreement, Nurix is deploying its proprietary DEL-AI drug discovery platform to identify novel agents that use E3 ligases to induce degradation of specified drug targets. Sanofi has an option to license drug candidates resulting from the work, and Nurix retains its option to co-develop and co-promote up to two future products in the United States after studies to assess dosing, efficacy, and safety that provide clinical proof of concept. For those programs for which Nurix exercises its option to co-develop and co-promote, the parties will split U.S profits and losses evenly and Nurix will be eligible to receive royalties on ex-U.S. sales on all optioned products. For programs that Nurix does not exercise its option, Nurix will receive milestones and royalties based on global development and sales. Upon signing the agreement in December 2019, Sanofi made an upfront payment of $55 million and subsequently paid an additional $22 million one year later to expand the scope of the collaboration. To date, Nurix has received a total of $127 million from Sanofi as part of this collaboration and remains eligible for up to $465 million in development, regulatory, and commercial milestones per licensed program as well as royalties on future sales.

About STAT6

STAT6 is a key transcription factor within the IL-4/IL-13 signaling pathways which act as drivers of inflammation in allergic conditions. Degrading STAT6 as a therapeutic strategy is supported by insights from mouse and human genetic studies as well as through clinical validation with either biologics targeting IL4/13 or small molecule inhibitors targeting the Janus Kinase (JAK) family. JAK proteins, which are upstream of STAT6, mediate the signaling of multiple cytokines, and as a result, JAK inhibition is associated with safety concerns. A potent, selective, and orally administered STAT6 degrader offers the potential for antibody-like efficacy and safety, with the convenience of a pill.

NuCana Reports First Quarter 2025 Financial Results and Provides Business Update

On June 2, 2025 NuCana plc (NASDAQ: NCNA) reported financial results for the first quarter ended March 31, 2025 and provided an update on its clinical development program with its two lead anti-cancer medicines (Press release, Nucana, JUN 2, 2025, View Source [SID1234653616]).

"We have entered 2025 with a clear focus on the advancement of our pipeline through key milestones, into late-stage development, and towards commercialization," said Hugh S. Griffith, NuCana’s Founder and Chief Executive Officer. "Our lead program, NUC-7738, continues to show significant promise. NUC-7738 is a novel agent that profoundly impacts gene expression in cancer cells and targets multiple aspects of the tumor microenvironment. In our ongoing Phase 1/2 NuTide:701 study, we have observed a favorable safety profile, meaningful tumor volume reduction and prolonged progression free-survival in patients with PD-1 inhibitor refractory and resistant metastatic melanoma. Based on these compelling results, we have recently initiated an expansion trial of NUC-7738 in an additional 28 patients with PD-1 inhibitor-resistant melanoma, that supports our registrational path. Based on this, we plan to meet with the U.S. Food and Drug Administration to determine the optimal regulatory strategy forward towards commercialization."

Mr. Griffith continued, "Turning to our second program, NUC-3373 is a targeted thymidylate synthase inhibitor with immune modulating properties. We are encouraged by the data from the ongoing Phase 1b/2 NuTide:303 study. This study is evaluating NUC-3373 in combination with pembrolizumab in patients with advanced solid tumors, and NUC-3373 with docetaxel in patients with lung cancer. To date, we have seen notable tumor volume reductions and prolonged progression free survival in these patients. We look forward to sharing additional data from this trial later this year."

Mr. Griffith concluded, "Lastly, we strengthened our balance sheet with a financing in May, extending our cash runway through key value-driving milestones. With multiple data readouts ahead, we are well-positioned to deliver on our mission of improving treatment outcomes for patients with cancer."

2025 Anticipated Milestones

NUC-7738

Initiate an expansion of the Phase 1/2 study (NuTide:701) of NUC-7738 in combination with pembrolizumab in patients with PD-1 inhibitor-resistant melanoma;

Announce data from the Phase 1/2 expansion study (NuTide:701) of NUC-7738 in combination with pembrolizumab; and

Obtain regulatory guidance from the U.S. Food and Drug Administration on pivotal study design for NUC-7738 in melanoma.

NUC-3373

Announce additional data from the Phase 1b/2 modular study (NuTide:303) of NUC-3373 in combination with pembrolizumab in patients with solid tumors.

First Quarter 2025 Financial Highlights and Cash Position

As of March 31, 2025, NuCana had cash and cash equivalents of £4.0 million compared to £6.7 million at December 31, 2024. Subsequent to March 31, 2025, NuCana completed a financing, raising an additional £8.8 million in gross proceeds before expenses and commission. NuCana expects that its cash and cash equivalents as of March 31, 2025, together with amounts raised through its financing, will be sufficient to fund its planned operations into Q4 2026.

NuCana continues to advance its numerous clinical programs and reported a net loss of £2.5 million for the quarter ended March 31, 2025, as compared to a net loss of £6.8 million for the quarter ended March 31, 2024. Basic and diluted loss per ordinary share was £0.02 for the quarter ended March 31, 2025, as compared to £0.13 per ordinary share for the comparable quarter ended March 31, 2024.

Mersana Therapeutics Reports Additional Positive Interim Phase 1 Clinical Data
for Emi-Le in Oral Presentation at 2025 ASCO Annual Meeting

On June 2, 2025 Mersana Therapeutics, Inc. (NASDAQ: MRSN), a clinical-stage biopharmaceutical company focused on the development of antibody-drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, reported additional interim Phase 1 clinical data for emiltatug ledadotin (Emi-Le; XMT-1660), the company’s B7-H4-directed Dolasynthen ADC (Press release, Mersana Therapeutics, JUN 2, 2025, View Source [SID1234653615]). These data are being presented in an oral session today at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2025 Annual Meeting in Chicago, Illinois.

The presentation will focus on Emi-Le’s Phase 1 dose escalation and backfill cohorts as of a March 8, 2025 data cut-off in patients with triple-negative breast cancer (TNBC); hormone-receptor-positive, human epidermal growth factor receptor 2-negative breast cancer; ovarian cancer; endometrial cancer and adenoid cystic carcinoma type 1 (ACC-1).

"We are excited to share additional interim data in an oral presentation at ASCO (Free ASCO Whitepaper)," said Martin Huber, M.D., President and Chief Executive Officer of Mersana Therapeutics. "The results from our dose escalation and backfill cohorts led us to focus our initial expansion work on patients with TNBC who have previously been treated with a topoisomerase-1 inhibitor ADC, a population with high unmet need that we believe Emi-Le may be uniquely suited to address within the B7-H4 ADC field. We also continue to be encouraged by the activity observed in other B7-H4-expressing tumor types, demonstrating Emi-Le’s broader development potential. On behalf of the Mersana team, we would like to express our gratitude to the patients and investigators who have participated in this clinical trial."

Safety and tolerability as of the March 8, 2025 data cut-off were consistent with initial data previously reported in January 2025, and no new safety signals were observed. Additionally, the following clinical activity data are being presented from evaluable patients (those with measurable disease at baseline and at least one post-baseline scan):

· 31% confirmed objective response rate (ORR) (8/26) across all enrolled tumor types with B7-H4 high tumor expression (defined as a tumor proportion score of 70% or higher) receiving intermediate Emi-Le doses (38.1 milligrams per meter squared (mg/m2) to 67.4 mg/m2 per cycle).

o 44% confirmed ORR (7/16) in the subset of patients with ≤4 prior lines of therapy.

· 56% ORR (5/9, including one unconfirmed response as of the March 8, 2025 data cut-off that was subsequently confirmed) in ACC-1 regardless of dose and B7-H4 expression. Among all ACC-1 patients who were enrolled in the Phase 1 clinical trial, the median progression free survival (PFS) had not yet been reached as of the March 8, 2025 data cut-off. ACC-1 is a rare and aggressive cancer that most frequently originates in the salivary glands. With no approved therapies, median PFS and median overall survival for patients with ACC-1 are reported to be 2-3 months and 2-3 years, respectively.

"The clinical activity observed for emiltatug ledadotin across all enrolled tumor types is encouraging, and its safety and tolerability profile appears differentiated from many other ADCs," said Antonio Giordano, MD, Ph.D., Assistant Professor of Medicine, Harvard Medical School, Dana-Farber Cancer Institute. "The objective responses were particularly notable in patients with late-stage triple-negative breast cancer who were previously treated with topoisomerase-1 inhibitors and in patients with ACC-1, both of which are aggressive and difficult-to-treat cancers with high unmet need."

The 2025 ASCO (Free ASCO Whitepaper) Annual Meeting data presentation can be accessed on the Publications section of the Mersana website at www.mersana.com.

About Emi-Le

Emi-Le is a B7-H4-directed Dolasynthen ADC with a precise, target-optimized drug-to-antibody ratio (DAR 6) and a proprietary auristatin payload with controlled bystander effect. This candidate is being investigated in Mersana’s ongoing Phase 1 clinical trial. The dose expansion portion of the Phase 1 clinical trial is enrolling patients with triple-negative breast cancer (TNBC) who have received one to four prior treatment lines, including at least one topoisomerase-1 inhibitor (topo-1) ADC.

The U.S. Food and Drug Administration has granted two Fast Track designations to Emi-Le for the treatment of 1) adult patients with advanced or metastatic triple-negative breast cancer, and 2) advanced or metastatic HER2 low / HER2 negative breast cancer post-topo-1 ADC (including TNBC and certain HR+ breast cancers). For more information about Mersana’s ongoing Phase 1 trial of Emi-Le, please visit clinicaltrials.gov (NCT05377996).

Kura Oncology and Kyowa Kirin Report Positive Pivotal Ziftomenib Monotherapy Data at 2025 ASCO Annual Meeting

On June 2, 2025 Kura Oncology, Inc. (Nasdaq: KURA, "Kura") and Kyowa Kirin Co., Ltd. (TSE: 4151, "Kyowa Kirin") reported the presentation of positive pivotal results from the KOMET-001 Phase 2 registration-directed trial of ziftomenib, a once-daily, oral investigational menin inhibitor, in patients with relapsed/refractory (R/R) NPM1-mutant (NPM1-m) acute myeloid leukemia (AML) in an oral session today at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held in Chicago, IL from May 30 – June 3, 2025 (Press release, Kura Oncology, JUN 2, 2025, View Source [SID1234653614]).

"We are delighted to announce positive pivotal data from the KOMET-001 trial in R/R NPM1-mutated AML patients treated with ziftomenib," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "NPM1 mutations are among the most common in AML, representing approximately 30% of cases, and there are no FDA-approved therapies specifically for this patient population. With these encouraging results and a PDUFA target action date of November 30, 2025, we and our partners at Kyowa Kirin look forward to supporting FDA with its review of the ziftomenib New Drug Application (NDA) and are well-positioned to meaningfully impact relapsed or refractory patients with NPM1 mutations."

"Relapsed or refractory NPM1-mutated AML is a highly challenging disease with a poor prognosis and an urgent need for new treatments," said Eunice Wang, M.D., Chief of Leukemia Service, Professor of Oncology, Roswell Park Comprehensive Cancer Center, Buffalo, NY. "The promising results for ziftomenib in this heavily pretreated population are highly encouraging. Notably, the clinically meaningful minimal residual disease (MRD)-negative responses observed as well as the similar response rates seen regardless of prior therapies, including hematopoietic stem cell transplantation (HSCT) and venetoclax, hold great promise for the potential use of ziftomenib in patients with relapsed and refractory NPM1-mutated AML."

The KOMET-001 Phase 2 population included 92 adult patients with R/R NPM1-m AML. The median age was 69 (range: 33 to 84). Patients were heavily pretreated, with 33% having received three or more prior lines of therapy (median prior lines: 2) and 59% having been previously treated with venetoclax.

A complete remission (CR) plus CR with partial hematological recovery (CRh) rate of 23% (21/92) was observed among patients with R/R NPM1-m AML in the Phase 2 portion of the KOMET-001 trial. Among those 21 patients who achieved CR/CRh, 13 had a CR and 8 had a CRh. The median duration of CR/CRh responses was 3.7 months (95% CI: 1.9, not estimable (NE)) and the restricted mean duration of response was 4.3 months (95% CI: 3.1, 5.6) at the time of the data cutoff. MRD status was assessed in 19 of 21 patients who achieved CR/CRh, and 63% (12/19) of these patients were MRD-negative.

Comparable CR/CRh rates were observed across pre-specified subgroups, regardless of prior HSCT, prior venetoclax or FLT3/IDH co-mutations. Additional patient benefit beyond CR/CRh was observed with a rate of transfusion conversion of 21% (17/82; 95% CI: 13-31) and a rate of maintenance of transfusion independence of 20% (2/10; 95% CI: 3-56). A median OS of 16.4 months (95% CI, 9.6–20.4) was observed for responders (patients who achieved CR, CRh, CRi/CRp, MLFS or PR) and a median overall survival (OS) of 3.5 months (95% CI, 2.5–4.0) was observed among non-responders.

The safety population included 112 adult patients with R/R NPM1-m AML from the pooled Phase 1b and Phase 2 portions of the KOMET-001 trial. The safety profile observed with ziftomenib in this population was consistent with previously reported data. Treatment-related adverse events (TRAEs) led to treatment discontinuations in 3% of patients. TRAEs of Grade ≥3 which occurred in more than 10% of patients were limited to differentiation syndrome (DS, 13%), which was well managed by protocol-specified mitigation strategies and no Grade 4/5 treatment-related DS was observed. Although QTc prolongation (1 Gr2; 2 Gr3) was reported in three patients per investigator assessment, all three patients were on concomitant medications associated with QTc prolongation, two had electrolyte abnormalities and one had a prior diagnosis of atrial fibrillation.

"Beyond ziftomenib’s clinical activity, we are highly encouraged by its consistent safety and tolerability profile," said Mollie Leoni, MD, Chief Medical Officer of Kura Oncology. "Notably, the low rate of myelosuppression, low discontinuation rate, lack of clinically significant QTc prolongation, absence of drug-drug interactions, and effective management of differentiation syndrome underscore ziftomenib’s potentially favorable benefit-risk profile for patients with relapsed or refractory NPM1-mutated AML."

"The data presented at ASCO (Free ASCO Whitepaper) strengthen our conviction that ziftomenib has potential to become a meaningful treatment option for patients with relapsed or refractory AML with NPM1 mutations — patients who often face limited treatment options and significant uncertainty regarding their prognosis," said Takeyoshi Yamashita, Ph.D., Executive Vice President and Chief Medical Officer of Kyowa Kirin. "Encouraged by the favorable safety, tolerability, and promising clinical activity observed thus far, Kyowa Kirin, in collaboration with Kura, is working with urgency and purpose to bring ziftomenib monotherapy to patients as swiftly and responsibly as possible."

Virtual Investor Event

Kura will host a virtual investor event featuring company management and investigators from the KOMET-001 trial of ziftomenib in R/R NPM1-m AML at 7:30pm ET / 4:30pm PT on Monday, June 2, 2025. Those who would like to participate may access the live webcast here, or register in advance for the teleconference here.The event can also be accessed on the Investors section of Kura’s website at www.kuraoncology.com. An archived replay will be available shortly after the conclusion of the live event.