NEW YORK, July 04, 2025 (GLOBE NEWSWIRE) -- WHY: Rosen Law Firm, a global investor rights law firm, reminds purchasers of securities of Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) between February 27, 2025 and May 26, 2025, both dates inclusive (the “Class Period”), of the important August 11, 2025 lead plaintiff deadline. SO WHAT: If you purchased Rocket Pharmaceuticals securities during the Class Period you may be entitled to compensation without payment of any out of pocket fees or costs through a conti ...

Medicus Pharma Ltd. (NASDAQ: MDCX ) describes itself as 'a biotech/life sciences company focused on accelerating the clinical development programs of novel and disruptive therapeutic assets'. The company came about in October 2023 as the result of aI have been investing in the stock market since I was 17 years old, and over the 25+ years since I have learned the joy of compounding, the value of dividend reinvesting, and the principle that patient investing through good times and bad brings the greatest rewa ...

Key Takeaways AstraZeneca gained EU approval for Imfinzi to treat muscle-invasive bladder cancer in adults. Imfinzi showed a 32% reduced risk of disease progression or death in the NIAGARA phase III study. Imfinzi sales rose 16% in Q1 2025 to $1.26B, driven by lung and liver cancer treatment demand.AstraZeneca (AZN) announced that the European Commission has approved its blockbuster cancer drug, Imfinzi (durvalumab), for a bladder cancer indication in the European Union (EU).The regulatory body in Europe ...

OnKure Therapeutics Overview - OnKure is developing mutant-selective PI3Kα inhibitors, targeting the majority of PI3Kα-mutated cancers while preserving wild-type PI3Kα [6, 9, 11] - The company's cash and investments are expected to provide funding through multiple clinical milestones and runway into Q4 2026 [6, 55] - OKI-219 is a PI3KαH1047R mutant selective inhibitor being evaluated in a Phase 1 trial as a monotherapy and in combination with fulvestrant in breast cancer [6] OKI-219 Clinical Development - OKI-219 is in Phase 1 clinical trials (PIKture-01) with data update expected in 2H 2025 [6, 9] - PIKture-01 trial includes Part A (monotherapy) and Part B (Fulvestrant Combination) [39, 51] - Initial combination data with fulvestrant expected in 2H 2025 [52] - As of March 31, 2025, OnKure had approximately $96.7 million in cash and investments [55] PI3Kα and Market Opportunity - PI3Kα is the most frequently mutated oncogene [6, 10] - PI3KαH1047R is present in approximately 15% of breast cancers and 4% of all human cancers [15] - OKI-219 exhibits approximately 80X selectivity for PI3KαH1047R vs PI3KαWT [17, 27]

Key Highlights of SpringWorks Therapeutics - OGSIVEO (nirogacestat) is the first and only FDA-approved therapy for desmoid tumors[15] - GOMEKLI (mirdametinib) is the first and only FDA-approved therapy for both adults and children with NF1-PN[15] - SpringWorks Therapeutics expects profitability in the first half of 2026[10] - SpringWorks Therapeutics had $461.9 million in cash, cash equivalents, and marketable securities[132] OGSIVEO (Nirogacestat) for Desmoid Tumors - OGSIVEO generated $61.5 million in net revenue in Q4 2024 and $172.0 million in FY 2024[15] - Between October 2023 and October 2024, approximately 11,000 patients with desmoid tumor ICD-10 claims were identified[15] - Objective response rate (ORR) increased from 34.3% with up to 1 year of treatment to 45.7% with up to 4 years of treatment[48] GOMEKLI (Mirdametinib) for NF1-PN - GOMEKLI is approved for the treatment of adult and pediatric patients 2 years of age and older with NF1-PN[10] - The U S market includes approximately 30,000 adult and 10,000 pediatric patients with NF1-PN[15] - In clinical trials, GOMEKLI showed a confirmed overall response rate of 41% in adults (n=58) and 52% in pediatrics (n=56)[67]

Tovecimig Clinical Trial Results (COMPANION-002 Study) - The COMPANION-002 study is a Phase 2/3 registrational-intent study in patients with BTC who have received one prior line of therapy[9] - In the Intent-to-Treat population, the Overall Response Rate (ORR) for Tovecimig + Paclitaxel was 17.1% (19 out of 111 patients) compared to 5.3% (3 out of 57 patients) for Paclitaxel alone, with a two-sided p-value of 0.031[12] - The Complete Response (CR) rate in the Tovecimig + Paclitaxel arm was 0.9% (1 out of 111 patients), while the Partial Response (PR) rate was 16.2% (18 out of 111 patients)[12] - Stable Disease (SD) was observed in 44.1% of patients (49 out of 111) in the Tovecimig + Paclitaxel arm compared to 33.3% (19 out of 57) in the Paclitaxel arm[12] - Progressive Disease (PD) was observed in 16.2% of patients (18 out of 111) in the Tovecimig + Paclitaxel arm compared to 42.1% (24 out of 57) in the Paclitaxel arm[12] Tovecimig Development and Potential - Tovecimig is a bispecific antibody targeting DLL4 and VEGF-A, designed to disrupt tumor vessel formation and angiogenesis[6, 7] - The company anticipates top-line Phase 2/3 data for PFS, OS, and DoR in Q4 2025[25] - Tovecimig has the potential to become a standard of care in 2L BTC, with PFS, OS and DoR data expected in Q4 2025[18] Market and Unmet Needs - There are significant unmet needs in current treatments for BTC, with approximately 85% of 2L patients having limited treatment options[19, 20] - Incidence of BTC is significant, with an estimated ~23,000 cases annually[22] - Projected ~100,000 incidence of liver and intrahepatic bile duct cancer by 2040[23]

TNX-103 and PH-HFpEF - TNX-103 (Oral Levosimendan) is a small molecule NCE with a dual mechanism of action, acting as a venodilator and enhancing cardiac contractility[6, 104] - TNX-103 has U S patent protection for multiple methods of use out to the end of 2040 with potential for continuations[7, 105] - The addressable market opportunity for TNX-103 is estimated to be at least $10 billion[9, 107] - Phase 3 program design aligned with the FDA helps de-risk the potential approval pathway for TNX-103[9, 107] Clinical Trial Data - In the Phase 2 HELP trial, TNX-103 showed statistically significant increases in 6MWD and reduction in PCWP across all exercise stages for PH-HFpEF patients[9, 107] - The OLE portion of the Phase 2 trial showed that patients who transitioned from IV to oral levosimendan in a 6-week period experienced improvements in 6MWD (+7 meters), BNP/NT-proBNP and KCCQ scores[9, 107] - 85% of patients enrolled in the HELP study responded with a robust decrease (≥4mm Hg) in exercise PCWP[45] - Oral Levosimendan causes a rapid and sustained reduction in NT-proBNP vs Baseline, with a 32% to 38% reduction in 7 days in the PERSIST Trial[79, 80] Market and Prevalence - PH-HFpEF affects an estimated 2 2 million US patients projected in 2030[9, 107] - In 2030, there will be an estimated 2 2 million to 3 7 million PH-HFpEF patients in the US[18] - Approximately 50% of heart failure patients with preserved ejection fraction (HFpEF) have PH[101]

PBFT02 Development and Preclinical Results - PBFT02 is an AAV gene therapy designed to deliver functional PGRN to the brain for the treatment of FTD-GRN [13] - In Grn-/- mice, AAV.hGRN vector ICV administration improved lysosomal function, reduced lipofuscin fluorescence in the thalamus, and reduced brain hexosaminidase activity [21, 23] - AAV1 was selected as the vector serotype due to superior hPGRN levels in CSF compared to AAV5 and AAVhu68 in NHPs [28, 29] - In Grn-/- mice, PBFT02 reduced lipofuscin deposition and neuroinflammation in the brain after intra-CSF delivery [34, 37] - ICM administration of PBFT02 enables PGRN delivery throughout the CNS [40] - In NHPs, PBFT02 dose-dependently increased PGRN in CSF up to day 14 [46, 48] - In NHPs, PBFT02 at Dose 1 resulted in approximately 10e4 GC/ug DNA throughout the brain [43] Clinical Trial (upliFT-D) and Safety - The upliFT-D trial is a global Phase 1/2 multi-center, open-label, dose-escalation study with PBFT02 [52, 55] - FTD-GRN Cohort 1 (n = 5) dosing is complete [56] - All four Cohort 1 participants who received a revised immunosuppression regimen had no SAEs or significant immune responses [57] - Cohort 1 interim data shows PBFT02 administration leads to robust and sustained increases in CSF PGRN [58]

Pipeline Highlights - ONCT-534, a Dual-Action Androgen Receptor Inhibitor (DAARI), is in Phase 1/2 clinical study for prostate cancer, with initial data expected in Q3 2024[8, 86] - ONCT-808, an autologous CAR T cell therapy targeting ROR1, is in Phase 1/2 clinical study for aggressive B-cell NHL, with a clinical data update expected in Q3 2024[8, 86] - Zilovertamab, a monoclonal antibody targeting ROR1, is seeking partnerships for further clinical trials in hematological malignancies and solid tumors[15, 81] ONCT-534 Key Points - The sixth cohort (1200 mg once daily) is fully enrolled in the Phase 1/2 dose escalation study in R/R mCRPC[8, 90] - ONCT-534 has shown activity in preclinical prostate cancer models of androgen receptor inhibitor resistance[8, 90] ONCT-808 Key Points - In the ONCT-808-101 study, 2 out of 3 patients achieved complete metabolic response (CMR) and 1 out of 3 patients achieved partial response (PR) in the 1x10^6 CAR T cells/kg cohort[72] - ONCT-808 CAR T cells expand and are persistent in all three patients from the 1 x 10^6 CAR T cells/kg dose cohort and the first patient from the 03 x 10^6 CAR T cells/kg dose cohort[75] Zilovertamab Key Points - In a pooled analysis with a median follow-up of 40 months, PFS for p53 mut/del(17p) was 100% for zilovertamab + ibrutinib[79, 80] Financial Highlights - As of March 31, 2024, Oncternal Therapeutics had $270 million in cash and short-term investments, providing a cash runway into Q1 2025[8, 87, 90] - The company anticipates $40 million in non-dilutive support through NIH grants[87]

Clinical Trial Results - The SIGNAL-AA Phase 2a trial studied bempikibart in patients with severe or very severe Alopecia Areata [1] - The study included patients with SALT scores between 50 and 100 [15] - At Week 24, patients with baseline SALT 50-100 showed a mean SALT score change of 163% with bempikibart treatment [20] - In patients with baseline SALT 50-95, the mean SALT score change at Week 24 was 245% with bempikibart [20] - Some patients experienced continued response even 7 months post last dose [23, 24] Safety and Tolerability - In the bempikibart group, 70% of participants experienced at least one treatment-emergent adverse event (TEAE) [27] - In the placebo group, 38% of participants experienced at least one TEAE [27] - No Grade 3 or higher related adverse events were reported in the bempikibart group [27] Study Design and Demographics - The study randomized patients in a 3:1 ratio to bempikibart (n=33) or placebo (n=81) [15] - The mean baseline SALT score in the bempikibart group was 749, while in the placebo group it was 819 [16]