On April 10, 2025 Pasithea Therapeutics Corp. (NASDAQ: KTTA) ("Pasithea" or the "Company"), a clinical-stage biotechnology company developing PAS-004, a next-generation macrocyclic MEK inhibitor, for the treatment of neurofibromatosis type 1 (NF1) and other cancer indications, reported that the external Safety Review Committee recommended that the Company’s Phase 1 clinical trial of PAS-004 in advanced cancer should proceed to Cohort 6, 30mg capsule, without modification (Press release, Pasithea Therapeutics, APR 10, 2025, View Source [SID1234651872]). This recommendation was based on the review of the safety data from three patients from Cohort 5 and the absence of any dose limiting toxicities (DLT’s). In addition, no rash has been observed to date during the DLT period in any of the first 19 patients in either capsule (15 patients) or tablet (four patients) formulation of PAS-004. Rash is a common adverse event (AE) that is observed at low doses with competitor MEK inhibitors and may lead to the discontinuation rate in real world practice.

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"We are seeing substantial enrollment demand and have already identified Cohort 6 patients. In addition, we continue to observe substantial exposure levels of PAS-004, and remain excited about the possibility of delivering relevant pERK inhibition below the no observed adverse effect levels (NOAEL) as we modeled and observed during our previously conducted nine-month chronic toxicity studies. The on label rash rate for both approved MEKi for NF1 exceeds 80% which leads to patients discontinuing who otherwise should remain on treatment for longer periods of time", stated Dr. Tiago Reis Marques, Chief Executive Officer of Pasithea. "We will provide additional safety, pharmacokinetic (PK) and pharmacodynamic (PD) data over the next several weeks."

The ongoing Phase 1 clinical trial is a multi-center, open-label, dose escalation 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).

On April 10, 2025 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported that two presentations and a panel discussion highlighting the ongoing Phase 2 open-label ADVANCED-2 trial of TARA-002 in patients with non-muscle invasive bladder cancer (NMIBC) will be featured at the upcoming American Urological Association (AUA) 2025 Annual Meeting taking place from April 26, 2025 to April 29, 2025 in Las Vegas (Press release, Protara Therapeutics, APR 10, 2025, View Source [SID1234651873]).

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A copy of the abstract for the ADVANCED-2 interim analysis is now available on the AUA website. Updated safety and efficacy data, including data from patients who have reached the 12-month evaluation timepoint, will be featured during an interactive poster session on Saturday, April 26, 2025.

ADVANCED-2 (NCT05951179) is a Phase 2 open-label trial assessing intravesical TARA-002 in NMIBC patients with carcinoma in situ or CIS (± Ta/T1) who are Bacillus Calmette-Guérin (BCG)-unresponsive (n≈100) and BCG-Naïve (n=30). The BCG-Unresponsive cohort has been designed to be registrational in alignment with the U.S. Food and Drug Administration’s 2024 BCG-Unresponsive Non-muscle Invasive Bladder Cancer: Developing Drugs and Biological Products for Treatment, Draft Guidance for Industry.

Presentation Details:

Title: Preliminary Anti-Tumor Activity and Safety Results from ADVANCED-2: A Phase 2 Open-Label Study of Intravesical TARA-002 in Adults with High-Grade Non-Muscle Invasive Bladder Cancer
Session: Bladder Cancer: Non-Invasive I
Presenter: Gautam Jayram, M.D., Director, Advanced Therapeutics Center, Urology Associates P.C., Nashville, TN
Session Date and Time: Saturday, April 26, 2025, 7:00 a.m. – 9:00 a.m. PT
Location: Marco Polo 701
Title: ADVANCED-2: A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of Intravesical Instillation of TARA-002 in Adults with High-Grade Non-Muscle Invasive Bladder Cancer
Session: Clinical Trials in Progress: Bladder Cancer
Presenter: Brian Mazzarella, M.D., Vice President of Research at Urology America, Austin, TX
Session Date and Time: Monday, April 28, 2025, 9:16 a.m. – 9:24 a.m. PT
Location: Hall C, The Square, Learning Lab
AUA Learning Lab Featured Trials Panel Discussion Details:

Title: ADVANCED-2: A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of Intravesical Instillation of TARA-002 in Adults with High-Grade Non-Muscle Invasive Bladder Cancer
Moderator: Jacqueline Zummo, Ph.D., Co-Founder, Senior Vice President, Chief Scientific Operations Officer, Protara Therapeutics
Speakers:
Timothy D. Lyon, M.D., Associate Professor of Urology and Urology Residency Program Director at Mayo Clinic, Jacksonville, FL
Brian Mazzarella, M.D., Vice President of Research at Urology America, Austin, TX
Alex Sankin, M.D., MS, Director of Clinical Trials Program, Associate Program Director of Urology Residency, Associate Professor and Attending Physician at Montefiore Medical Center, Bronx, NY
Date and Time: Monday, April 28, 2025, 11:00 a.m. – 11:30 a.m. PT
About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and of LMs, for which it has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil in Japan by Chugai Pharmaceutical Co., Ltd and also approved in Taiwan. Protara has successfully shown manufacturing comparability between TARA-002 and OK-432.

When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a pro-inflammatory response with release of cytokines such as tumor necrosis factor (TNF)-alpha, interferon (IFN)-gamma IL-6, IL-10, IL-12. TARA-002 also directly kills tumor cells and triggers a host immune response by inducing immunogenic cell death, which further enhances the antitumor immune response.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

Bladder cancer is the 6th most common cancer in the United States, with NMIBC representing approximately 80% of bladder cancer diagnoses. Approximately 65,000 patients are diagnosed with NMIBC in the United States each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle.

On April 9, 2025 Halozyme Therapeutics, Inc. (NASDAQ: HALO) (Halozyme) reported that Janssen-Cilag International NV, a Johnson & Johnson company, received European Commission (EC) approval for an indication extension of DARZALEX (daratumumab) subcutaneous (SC) co-formulated with ENHANZE in the frontline setting (Press release, Halozyme, APR 9, 2025, View Source [SID1234651857]). The approval is for daratumumab SC in combination with bortezomib, lenalidomide, and dexamethasone (daratumumab-VRd) for the treatment of adult patients with newly diagnosed multiple myeloma.1

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"The continued expansion of DARZALEX delivered subcutaneously with ENHANZE into additional settings highlights its status as a cornerstone of therapy for multiple myeloma," said Dr. Helen Torley, President and CEO of Halozyme. "This approval means that newly diagnosed patients can receive daratumumab subcutaneous plus VRd and avoid the need for lengthy IV infusions."

This approval follows the indication extension approval for daratumumab-VRd in October 2024, for the treatment of newly diagnosed patients with multiple myeloma who are eligible for autologous stem cell transplant, based on the results from the Phase 3 PERSEUS (NCT03652064) study. The study evaluated this daratumumab SC-based quadruplet regimen for induction and consolidation therapy, followed by daratumumab SC and lenalidomide maintenance.2,3

1 European Medicines Agency. DARZALEX (daratumumab) Summary of Product Characteristics. April 2025.

2 Rodríguez-Otero P, et al. Daratumumab (DARA) + bortezomib/lenalidomide/dexamethasone (VRd) in transplant-eligible (TE) patients (pts) with newly diagnosed multiple myeloma (NDMM): Analysis of minimal residual disease (MRD) in the PERSEUS trial. 2024 American Society for Clinical Oncology Annual Meeting. June 3, 2024.

3 Johnson & Johnson Innovative Medicine EMEA. DARZALEX (daratumumab)-SC based quadruplet regimen approved by the European Commission for patients with newly diagnosed multiple myeloma who are transplant-eligible. Available at: View Source Last accessed: April 2025.

On April 9, 2025 Harbour BioMed (HKEX: 02142), a global biopharmaceutical company committed to the discovery and development of novel antibody therapeutics focusing on immunology and oncology, reported the appointment of Youchen (YC) Chen as Chief Financial Officer (Press release, Harbour BioMed, APR 9, 2025, View Source [SID1234651858]). YC will be based in Shanghai and Hong Kong SAR, and report directly to Dr. Jingsong Wang, Founder, Chairman, and CEO of Harbour BioMed.

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Since joining Harbour BioMed in 2023, YC has taken on increasing responsibilities across Investor Relations, Corporate Development, Business Development, and Finance. His strategic vision and collaborative leadership have significantly contributed to key strategic transactions, global partnerships and alliances, as well as global financial management and operations. In his new role, YC will continue to leverage his expertise to further enhance shareholder value and guide Harbour BioMed through its next phase of strategic development.

Before joining Harbour BioMed, YC served as Chief Financial Officer at a clinical-stage radiopharmaceutical startup. He was previously Vice President at Credit Suisse China Investment Banking and Capital Market team, advising healthcare and technology companies in Greater China on strategic and financing transactions. Prior to Credit Suisse, YC held multiple roles as Head of Corporate Finance at Yidu Tech (2158.HK) and Audit Assistant Manager at KPMG.

Dr. Jingsong Wang, Founder, Chairman, and CEO of Harbour BioMed, commented: "YC has brought a strong strategic mindset and deep financial expertise to Harbour BioMed. His ability to lead cross-functional initiatives and drive value creation has made a significant impact across the organization. I’m confident that in his new role as Chief Financial Officer, he will continue to strengthen our financial foundation and support our long-term growth."

Youchen Chen, Chief Financial Officer of Harbour BioMed, added: "I’m honored to take on the role of Chief Financial Officer at Harbour BioMed. It’s been a privilege to work alongside such a dedicated and visionary team. I look forward to continuing our efforts to advance the company’s strategic objectives, deliver value to our stakeholders, and support our mission of transforming innovation into impactful therapies."

YC holds a bachelor’s degree in law from Fudan University and an MBA from the McDonough School of Business at Georgetown University. He is also currently pursuing an advanced degree at Harvard Medical School.

On April 9, 2025 Engine Biosciences (Engine), a Singapore- and Silicon Valley-based biotechnology company pioneering precision medicine for cancer, reported a new partnership with the Experimental Drug Development Centre (EDDC), Singapore’s national platform for drug discovery and development hosted by the Agency for Science, Technology and Research (A*STAR) (Press release, Experimental Drug Development Centre, APR 9, 2025, View Source [SID1234654013]).

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This first-of-its-kind collaboration unites Engine’s NetMAPPR platform and proprietary oncology intellectual property with EDDC’s drug discovery and development expertise to create first-in-class precision cancer treatments.

The first project under this partnership focuses on ENB-871, a novel pairing of a drug target and patient selection biomarkers discovered through Engine’s NetMAPPR platform. This platform combines AI, computation, biology, and chemistry to identify, validate, and prioritise drug targets with strong clinical and commercial potential, driving the development of new therapies that exploit specific vulnerabilities in tumours.

This programme shows significant promise for treating tumours with particular genetic mutations that predict sensitivity to the ENB-871 targeted therapy, including breast, liver, kidney and prostate cancers – diseases afflicting large and growing patient populations in Singapore and worldwide. In total, the potential addressable population exceeds 500,000 patients per year.

The teams will collaborate to develop small molecule degraders targeting ENB-871, including demonstration of in vivo efficacy. By bringing together Engine’s proprietary technology and deep translational insights with EDDC’s strengths in designing and developing therapies, this partnership aims to create targeted cancer treatments tailored to patients’ specific profiles, improving treatment effectiveness and outcomes. Engine and EDDC may also identify additional drug targets and research programmes for collaboration during the partnership.

"We’re excited by the synergies created by bringing together our two platforms, leveraging first-in-class Singapore research and innovation to advance transformative cancer therapies. This marks another key step in Engine’s mission to develop more effective, targeted and safer drugs for cancer patients." said Jeffrey Lu, CEO and Co-Founder of Engine Biosciences.

"The future of drug development lies in precision-driven innovation. Our partnership with Engine enables us to develop therapies tailored to specific patient populations through Engine’s biomarker-driven patient selection approaches. We are particularly excited to launch our first collaborative project around monovalent small molecule degraders, building on EDDC’s expanding capabilities in this field. Beyond this, we look forward to strengthening our partnership by advancing more precision therapies that have the potential to transform the lives of cancer patients in need," shared Damian O’Connell, CEO of EDDC.