On May 14, 2024 ESSA Pharma Inc. ("ESSA", or the "Company") (NASDAQ: EPIX), a clinical-stage pharmaceutical company focused on developing novel therapies for the treatment of prostate cancer, reported a corporate update and announced financial results for the fiscal second quarter ended March 31, 2024 (Press release, ESSA, MAY 14, 2024, View Source [SID1234643269]).

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"The year is off to a strong start with the presentation of updated Phase 1 masofaniten dose escalation data at the 2024 ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium ("ASCO-GU"), which demonstrated that masofaniten combined with enzalutamide continues to be well tolerated with deep and durable reductions in prostate-specific antigen ("PSA") in patients with metastatic castration-resistant prostate cancer ("mCRPC") naïve to second-generation antiandrogens," said David Parkinson, MD, President and CEO of ESSA. "Looking ahead, we have multiple critical milestones we are working toward, including reporting more updated data from the Phase 1 dose escalation study evaluating masofaniten combined with enzalutamide in this patient population during the second half of 2024, and completing enrollment in the Phase 2 dose expansion study evaluating masofaniten in combination with enzalutamide during the first quarter of 2025, with preliminary data expected to follow in mid-2025."

Second Quarter Fiscal 2024 and Recent Highlights

Masofaniten Combination Studies

Reported updated Phase 1 dose escalation data from the ongoing Phase 1/2 study evaluating masofaniten in combination with enzalutamide in patients with mCRPC naïve to second-generation antiandrogens but may have been treated with chemotherapy in the metastatic castration-sensitive setting. The results, which were presented at the ASCO (Free ASCO Whitepaper)-GU symposium in January 2024, demonstrated that the combination regimen continues to be well tolerated at the dose levels tested in up to 25 cycles of dosing in some patients. Reductions in PSA were observed across evaluable patients for efficacy in all dosing cohorts (n=16). Across all dosing cohorts, 88% of patients achieved PSA50, 81% of patients achieved PSA90, 69% of patients achieved PSA90 in less than 90 days, and 63% of patients achieved PSA <0.2ng/mL. While the data for time to PSA progression were still maturing, the median time to PSA progression was reported as 16.6 months with a median follow up at that time of 11.1 months. ESSA expects to report updated data from the Phase 1 dose escalation study during the second half of 2024.
Masofaniten is currently being evaluated in combination with enzalutamide compared to enzalutamide monotherapy in a Phase 2 dose randomized study in patients with mCRPC naïve to second-generation antiandrogens but who may have been treated with chemotherapy in the metastatic castration-sensitive setting. Enrollment in the Phase 2 portion of this Phase 1/2 study is expected to be completed during the first quarter of 2025. This Phase 2 study has an open-label randomized design comparing 160 mg once-daily of single agent enzalutamide to the combination of masofaniten with enzalutamide and is expected to enroll approximately 120 patients. The recommended Phase 2 combination dose was identified as masofaniten 600 mg twice-daily combined with enzalutamide 160 mg once daily. The study is currently enrolling at approximately 25 sites in the USA, Canada, and Australia. Expansion to European clinical sites is in progress with an additional 15 clinical sites planned to be activated by 3Q24. ESSA expects to report preliminary data from the Phase 2 dose expansion portion of the study in mid-2025.
Two additional masofaniten combination arms are currently enrolling as part of the ongoing Phase 1 masofaniten study. One arm is evaluating masofaniten in combination with abiraterone acetate and prednisone in patients with either metastatic castration-sensitive prostate cancer or mCRPC, while the second arm is evaluating masofaniten in combination with apalutamide in patients with non-metastatic castration-resistant prostate cancer after 12 weeks of masofaniten single agent.
Two additional investigator-sponsored studies testing combinations of masofaniten with darolutamide or enzalutamide in different patient populations are underway: a) an Australian investigator-sponsored neoadjuvant study evaluating neoadjuvant use of the combination of masofaniten and darolutamide compared to darolutamide monotherapy in high-risk patients undergoing prostatectomy and b) an investigator-sponsored study which is testing masofaniten and enzalutamide in metastatic castration-sensitive prostate cancer patients.
Masofaniten Monotherapy Study

ESSA remains on track to complete the Phase 1b masofaniten monotherapy study evaluating masofaniten in patients with mCRPC resistant to second-generation antiandrogens. The initial results from the monotherapy study were reported at the 2023 ASCO (Free ASCO Whitepaper)-GU Symposium, and demonstrated that masofaniten monotherapy was well-tolerated, achieved clinically significant exposures, and showed preliminary signals of anti-tumor activity in a subset of patients. ESSA plans to present the complete Phase 1a and 1b monotherapy results in the second half of 2024 at a medical conference.
Summary Financial results
(Amounts expressed in U.S. dollars)

Net Loss. ESSA recorded a net loss of $9.0 million for the second quarter ended March 31, 2024 compared to $7.1 million for the second quarter ended March 31, 2023. The increase in the second quarter was primarily attributed to investment in the Company’s clinical trials.
Research and Development ("R&D") expenditures. R&D expenditures for the second quarter ended March 31, 2024 were $6.2 million compared to $4.5 million for the second quarter ended March 31, 2023, and include non-cash costs related to share-based payments $459,141 for the second quarter ended 2024 compared to $750,159 for the second quarter ended 2023. Increased enrollment in the clinical trials and expanded sites in the period resulted in higher levels of investment.
General and Administration ("G&A") expenditures. G&A expenditures for the second quarter ended March 31, 2024 were $4.3 million compared to $3.7 million for the second quarter ended March 31, 2023 and include non-cash costs related to share-based payments of $673,460 for the second quarter ended 2024 compared to $686,932 for the second quarter ended 2023. Professional fees were incurred for legal and accounting services in conjunction with ongoing corporate activities.
Liquidity and Outstanding Share Capital

As of March 31, 2024, the Company had available cash reserves and short-term investments of $135.9 million. The Company’s cash position is expected to be sufficient to fund current and planned operations beyond 2025.
As of March 31, 2024, the Company had 44,362,991 common shares issued and outstanding.
In addition, as of March 31, 2024, there were 2,920,000 common shares issuable upon the exercise of prefunded warrants at an exercise price of $0.0001.

On May 14, 2024 Nanopharmaceutics, Inc. (OTC:TGRP), a clinical-stage pharmaceutical development company, reported the initiation of a Phase I clinical study with the Robert H. Lurie Comprehensive Cancer Center of Northwestern University "A Phase 1 Adaptive Dose Escalation With Dose Expansion Study of Triapine in Combination With Temozolomide (TMZ) for Patients With Recurrent Glioblastoma" (ClinicalTrials.gov Identifier: NCT06410248) (Press release, Nanopharmaceutics, MAY 14, 2024, View Source [SID1234643268]). The primary objective of this phase 1 study will be to determine the recommended phase 2 dose (RP2D) for Triapine in combination with temozolomide (TMZ). Secondary objectives include evaluation of the safety profile of Triapine in combination with temozolomide (TMZ), progression-free survival (PFS), overall survival (OS), overall response rate (ORR) per RANO criteria, and quality of life per FACT-Br. The study will recruit 40-45 patients with an established diagnosis of recurrent glioblastoma, and there will be an exploratory arm to investigate drug delivery into brain tumor tissue and target engagement in a cohort who are surgical candidates for re-resection of recurrent glioblastoma. Funding for the study is provided by BrainUp.

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Oral Triapine has been shown to be safe, tolerable, and convenient in multiple clinical studies. Ribonucleotide Reductase (RNR) Regulatory Subunit 2 (RRM2) targeting with Triapine may overcome chemoresistance and improve the clinical efficacy of TMZ therapy. Preliminary research from Dr. Atique Ahmed’s lab at Northwestern University has uncovered a critical factor that helps glioblastoma, an aggressive brain cancer, resist chemotherapy treatment.

About the Robert H. Lurie Comprehensive Cancer Center of Northwestern University

The Robert H. Lurie Comprehensive Cancer Center of Northwestern University is a National Cancer Institute-designated Comprehensive Cancer Center located on Northwestern Memorial Hospital’s downtown medical campus in the Streeterville neighborhood of Chicago, Illinois, United States.

On May 14, 2024 Caris Life Sciences (Caris), the leading next-generation AI TechBio company and precision medicine pioneer, reported that the company and collaborators from the biopharma industry and leading cancer centers, including those within the Caris Precision Oncology Alliance (POA), will collectively present 41 studies across 19 tumor types at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting May 31 to June 4, 2024 (Booth #28001) (Press release, Caris Life Sciences, MAY 14, 2024, View Source [SID1234643267]). The findings demonstrate the power of Caris’ comprehensive multi-modal database to enable novel insights into cancer that could have profound effects on a patient’s diagnosis, prognosis, care plan and response to treatment.

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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

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"We are immensely proud of the collaborative studies accepted for presentation at ASCO (Free ASCO Whitepaper), which are a testament to the value of Caris’ comprehensive molecular profiling and the power of our ever-expanding network of POA collaborations," said Caris EVP and Chief Medical Officer George W. Sledge, Jr., MD. "The diverse array of findings underscores the critical role of comprehensive profiling in cancer care and the power of large clinico-genomic datasets to enable the identification of new biomarkers with clinical implications across diverse tumor types, including lung, prostate and breast cancer. Through extensive sequencing and data analysis efforts, we’re unlocking new avenues for tailored therapies to revolutionize cancer treatment."

"Caris enables clinicians to make the best individualized treatment choices for their patients, researchers to discover new actionable targets and our biopharmaceutical partners to develop the next breakthrough medicines," said Caris President David Spetzler, MS, PhD, MBA. "In collaboration with leading oncology experts in the POA, our clinicians and scientists leverage vast real-world evidence from over 632,000 lifetime clinical cases, including over 482,000 with matched molecular data and outcomes in Caris’ unique AI-driven platform, to unravel the complexities of cancer. Together, we’re revealing the intricacies of the molecular and immune landscapes of tumors and how these impact clinical outcomes, paving the way for personalized therapies and improved patient outcomes."

Rapid Oral Presentations:

Genomic and tumor microenvironment dynamics of brain metastases in breast cancer. (Abstract Number: 1018)
Monday, June 3; 12:06 PM – 12:10 PM CDT

Clinical utility of transcriptomic signatures to identify androgen receptor and neuroendocrine signaling in prostate cancer. (Abstract Number: 5015)
Monday, June 3; 2:03 PM – 2:09 PM CDT
Merit Award Poster:

The molecular landscape of pembrolizumab and lenvatinib treatment in endometrial cancer. (Abstract Number: 5612)
Monday, June 3; 9:00 AM – 12:00 PM CDT
Other notable studies focus on key topics including molecularly guided treatment strategies, predictors of therapeutic response and novel prognostic biomarkers:

The molecular landscape of PIWIL1 expression in colorectal adenocarcinoma (CRC). (Abstract Number: 3546)
Saturday, June 1; 1:30 PM – 4:30 PM CDT

RNA expression-based hypoxia score as a prognostic and predictive biomarker in hepatocellular carcinoma. (Abstract Number: 4026)
Saturday, June 1; 1:30 PM – 4:30 PM CDT

Therapeutic strategy for targeting recurrent oncogenic kinase gene fusions in prostate cancer. (Abstract Number: 5093)
Sunday, June 2; 9:00 AM – 12:00 PM CDT

Kinome reprogramming as a therapeutic opportunity in ESR1 fusion driven breast cancer but not in gynecologic cancers. (Abstract Number: 1045)
Sunday, June 2; 9:00 AM – 12:00 PM CDT

Molecular and immunological characterization of androgen receptor expression in different breast cancer subtypes. (Abstract Number: 1114)
Sunday, June 2; 9:00 AM – 12:00 PM CDT

Characterization of TP53 mutations in actionable driver-negative non-small cell lung cancer (NSCLC). (Abstract Number: 8621)
Monday, June 3; 1:30 PM – 4:30 PM CDT

Association of germline HSD3B1 (c.1100) genotype with tumoral and clinical outcomes in breast and endometrial cancers. (Abstract Number: 10587)
Monday, June 3; 1:30 PM – 4:30 PM CDT

Immunotherapy outcomes and profile in lung cancer brain metastases. (Abstract Number: 8646)
Monday, June 3; 1:30 PM – 4:30 PM CDT

Multidimensional analysis of B7 homolog 3 (B7-H3) RNA expression in small-cell lung cancer (SCLC) molecular subtypes. (Abstract Number: 8088)
Monday, June 3; 1:30 PM – 4:30 PM CDT
Poster and abstract summaries highlighting the Caris research presented at ASCO (Free ASCO Whitepaper) 2024 will be available onsite at Caris’ Booth 28001. The full abstracts will be available on the Caris website beginning on June 1st.

The POA includes 91 cancer centers, academic institutions, research consortia and healthcare systems, including 43 NCI-designated cancer centers, collaborating to advance precision oncology and biomarker-driven research. POA members work together to establish and optimize standards of care for molecular testing through innovative research focused on predictive and prognostic markers that improve the clinical outcomes for cancer patients.

On May 14, 2024 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a Cayman incorporated biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, reported financial results for the three months ended March 31, 2024 (Press release, CASI Pharmaceuticals, MAY 14, 2024, View Source [SID1234643266]).

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In the first quarter of 2024, CASI’s total revenue was $3.4 million. Total revenue decreased by 59% compared to the same period in 2023. This decline can be attributed to several factors: increasing competition from a generic melphalan product with lower cost and undifferentiated formulation, implementation of an inventory management strategy by the distributor of EVOMELA and restructuring of the sales force. CASI’s commercial and medical marketing team remain committed to executing a strategic plan and defending our market leadership position for EVOMELA. Furthermore, CASI has successfully launched its second commercial product, FOLOTYN, and administered the first patient dose in China in February. We will continue to spend time, resources, and efforts to grow our commercial franchise throughout 2024 and beyond.

Advancement, development, and commercialization of the pipelines remain our strategic focus. We continue to make progress on the development of BI-1206 in China. The early clinical data from the ongoing Phase I trial in China has demonstrated promising preliminary results for patients with relapsed/refractory non-Hodgkin lymphoma. We submitted an Investigational New Drug Application (IND) to the US Food and Drug Administration (FDA) for CID-103 on immune thrombocytopenia (ITP) in April of 2024. We are excited to initiate clinical development for autoimmune indications of this program in the US. CB-5339 received Clinical Trial Application approval from the NMPA in 2023. With respect to Inaticabtagene Autoleucel (CNCT-19 CAR-T cell therapy), we have received an emergency injunctive relief that prohibits Juventas from commercializing CNCT-19 by itself or through another third party, and while continuing to vigorously assert and enforce our rights with respect to the commercial launch of CNCT-19, we intend to negotiate with Juventas in good faith the temporary arrangement for new patients’ access to CNCT-19 treatment. We will continue to drive our portfolio forward by executing on several milestones in the quarters ahead.

First Quarter 2024 Financial Highlights

Total revenue was $3.4 million for the three months ended March 31, 2024, compared to $8.3 million for the three months ended March 31, 2023.

Costs of revenues were $1.6 million for the three months ended March 31, 2024, compared to $3.4 million for the three months ended March 31, 2023. The decrease was in line with the decrease of revenues.

Research and development expenses for the three months ended March 31, 2024, were $2.5 million, compared with $2.5 million for the three months ended March 31, 2023.

General and administrative expenses for the three months ended March 31, 2024, were $4.8 million, compared with $5.7 million for the three months ended March 31, 2023.

Selling and marketing expenses for the three months ended March 31, 2024, were $3.7 million, compared with $4.0 million for the three months ended March 31, 2023.

Net loss for the three months ended March 31, 2024, were $9.5 million, compared with $5.8 million for the three months ended March 31, 2023. The expanding of net loss was mainly attributed to the decrease in revenues.

As of March 31, 2024, CASI had cash, cash equivalents and short term investments of $18.2 million compared to $29.1 million as of December 31, 2023.

Further information regarding the Company, including its Quarterly Report for the quarter ended March 31, 2024, can be found at www.casipharmaceuticals.com.

On May 12, 2024 Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focusing on novel small molecule therapies that address emerging targets in oncology, reported that it will present clinical and preclinical data from RVU120 at the 2024 European Hematology Association (EHA) (Free EHA Whitepaper) Congress (EHA) (Free EHA Whitepaper), June 13-16, Madrid, Spain (Press release, Ryvu Therapeutics, MAY 14, 2024, View Source [SID1234643265]).

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"At this year’s EHA (Free EHA Whitepaper) conference, we proudly unveil the latest advancements in RVU120 research and clinical development, illuminating its potential in addressing the unmet need in hematologic malignancies. RVU120 has demonstrated clinical activity as a single agent in 15 of 29 evaluable patients with AML and HR-MDS at tolerated doses. Furthermore, our preclinical data demonstrate the synergistic potential of RVU120 in combination with venetoclax, representing a promising strategy against resistance in AML. We are also excited about the emerging data in MPN models, where combination regimens with RVU120 could represent a new treatment paradigm." – Dr. Hendrik Nogai, Chief Medical Officer, Ryvu Therapeutics.

Based on these data and in line with prior guidance, Ryvu’s development plan for RVU120 includes four Phase II studies: the RIVER-52 study in patients with AML/HR-MDS as monotherapy, the RIVER-81 study in combination with venetoclax in patients with r/r AML, the POTAMI-61 study in patients with myelofibrosis, and the REMARK study, an investigator-initiated trial in patients with LR-MDS.

Poster highlights:

Abstract Title: RVU120, a first-in-class CDK8 inhibitor for the treatment of relapsed/refractory AML and high-risk MDS: preliminary results from two ongoing studies.

Number: 6466
Session date and time: Friday, June 14 (18:00-19:00 CEST)

The abstract includes data on 29 evaluable patients out of 38 total dosed patients.

In the Phase I trial CLI120-001, RVU120 shows promising clinical activity in patients with AML or HR-MDS. 15 out of 38 patients experienced clinical benefit with RBC transfusion independence and/or blast reduction. A complete remission (CR) was achieved in a patient with NPM1 and DNMT3A mutations as well as 3 marrow CRs in patients with HR-MDS. Relevant target inhibition was achieved at a dose of 110 mg or higher, supporting a recommended Phase II dose (RP2D) of 250mg. The follow-on Phase II RIVER-52 study investigates RVU120 as a monotherapy in patients with genetically defined AML or HR-MDS and is currently recruiting.

Abstract Title: Synergistic potential of RVU120, a first-in-class CDK8/CDK19 inhibitor, with venetoclax in AML: preclinical and initial clinical insights.

Abstract Number: 6720
Session date and time: Friday, June 14 (18:00-19:00 CEST)

The abstract presents updated preclinical data supporting the synergistic combination of RVU120 and venetoclax in AML, including RVU120’s potential to overcome resistance to venetoclax treatment. Ongoing preclinical research and an ongoing Phase II clinical study seek to refine patient stratification and enhance therapeutic outcomes, with initial safety profiles appearing favorable. The Phase II RIVER-81 study investigates RVU120 in combination with venetoclax in patients with AML and is currently recruiting.

Abstract Title: CDK8/19 Inhibition: A Promising Therapeutic Strategy in Myeloproliferative Neoplasms.

Poster Number: P1018

Session date and time: Friday, June 14 (18:00-19:00 CEST)

The presentation, prepared in collaboration with Prof. Raajit Rampal’s group from Memorial Sloan Kettering Cancer Center, includes the assessment of RVU120 as a monotherapy and in combination with JAK inhibitors and other drug candidates for the treatment of myeloproliferative neoplasms (MPN). Analysis of the effects from combinations of RVU120 with other compounds in vitro has revealed potentially new therapeutic options for patients with MPN sensitivity and resistance to ruxolitinib (RUX). In vivo data further support CDK8 inhibition as a potential novel therapeutic strategy in MPNs. Additional mechanistic work will potentially elucidate disease mechanisms and therapeutic action independent of JAK-STAT attenuation. This work has led to a Phase II clinical study (POTAMI-61) for RVU120 as a monotherapy in patients with myelofibrosis patients not eligible for RUX or in combination with RUX for those patients with myelofibrosis and suboptimal RUX response.

The final posters that will be presented on June 14th will contain additional information.

Investor Event:

Ryvu will host a webinar on Friday, May 17, at 3:00 PM CET with Professor Raajit Rampal, who will present the myelofibrosis landscape, the latest data and the potential of RVU120 in MF.

To join the webcast, please register here: View Source