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Yahoo
03-06-2025
- Business
- Yahoo
Alphamab Oncology Presented Multiple Clinical Data of Anti-HER2 Biparatopic ADC JSKN003 at the 2025 ASCO Annual Meeting
SUZHOU, China, June 3, 2025 /PRNewswire/ -- Alphamab Oncology (stock code: announced that multiple clinical data updates for anti-HER2 biparatopic antibody-drug conjugate (ADC) JSKN003 were presented as posters at the 2025 Annual Meeting of American Society of Clinical Oncology (2025 ASCO Annual Meeting) from May 30 to June 3, 2025, in Chicago, IL, U.S.. The results covered platinum-resistant ovarian cancer, HER2-positive breast cancer, and HER2-overexpressing gastrointestinal tumors. Title: JSKN003, a biparatopic anti-HER2 antibody drug conjugate (ADC), in the treatment of platinum-resistant ovarian cancer (PROC): Updated findings from two clinical trialsAbstract Number for Publication: 5557Session Type and Title: Poster Session - Gynecologic CancerSession Date and Time: 6/1/2025 9:00 AM-12:00 PM CDTPresenter: Xiaohua Wu, Fudan University Shanghai Cancer Center Methods JSKN003-101 (NCT05494918) is a Phase I study in Australia and JSKN003-102 (NCT05744427) is a Phase I/II study in China. Both trials enrolled patients with advanced solid tumors who were to receive JSKN003 monotherapy at various dose levels. Pooled results have demonstrated that JSKN003 monotherapy has promising efficacy signals in patients with PROC, and the efficacy was observed across patients with (IHC 1+/2+/3+) or without (IHC 0) HER2 expression, with or without prior bevacizumab and prior PARP inhibitor. Preliminary data from the pooled analysis of these two studies were presented at the 2024 European Society for Medical Oncology (ESMO) Congress for the first time. The latest findings for non-primary platinum-refractory PROC patients at a longer follow-up time were reported at this ASCO Annual Meeting. Results As of February 28, 2025, 46 PROC patients were enrolled and received JSKN003 every three weeks across five doses levels, among which 2 patients at the dose of 4.2 mg/kg, 2 patients at the dose of 5.2 mg/kg, 40 patients at the dose of 6.3 mg/kg (RP2D), 1 patient at the dose of 7.3mg/kg, and 1 patient at the dose of 8.4mg/kg. Efficacy: With a median follow-up time of 9.3 months, 46 patients were efficacy evaluable. 42 patients (91.3%) exhibited tumor shrinkage. The objective response rate (ORR) was 63.0%, the median progression-free survival (PFS) was 7.7 months, and the 9-month overall survival (OS) rate was 89.9%. Efficacy was observed across different HER2-expression subgroups. The ORR was 52.4% and the median PFS was 6.6 months in patients with HER2 IHC 0. The ORR reached 72.2% and the median PFS was 9.4 months in patients with HER2 expression (IHC 1+/2+/3+). Safety: Grade 3-4 treatment-related adverse events (TRAEs) occurred in 9 patients (19.6%). Serious TRAEs were reported in 6 patients (13.0%). No TRAEs leading to death. Interstitial lung disease (ILD) was observed in 5 patients (10.9%), all were Grade 1/2. Conclusions With extended follow-up, JSKN003 demonstrated robust PFS improvement in PROC, along with early signals of OS benefit. A confirmatory trial (JSKN003-306, NCT06751485) is currently enrolling all comers regardless of HER2 expression to validate JSKN003 as a treatment option for this patient population. Title: JSKN003, a biparatopic HER2-targeting ADC, in heavily pretreated HER2-positive breast cancer: A pooled analysis of early-phase studiesAbstract Number for Publication: 1028Session Type and Title: Poster Session - Breast Cancer - MetastaticSession Date and Time: 6/2/2025 9:00 AM-12:00 PM CDTPresenter: Yiqun Du, Fudan University Shanghai Cancer Center Methods The pooled analysis was performed to evaluate the efficacy and safety of JSKN003 in HER2-positive (IHC 3+ or 2+/ISH+) advanced breast cancer from the Phase I clinical trial (JSKN003-101, NCT05494918) in Australia and the Phase I/II clinical trial (JSKN003-102, NCT05744427) in China. Results As of February 28, 2025, the median follow-up duration was 6.1 months. A total of 88 patients with HER2-positive breast cancer were enrolled, with the majority receiving 6.3 mg/kg or 8.4 mg/kg doses. The median age was 55 years (range: 32-79), with 77.3% ECOG PS 1. All patients had stage IV disease, with 76.1% having visceral metastases. All patients had prior anti-HER2 therapy, including 85.2% with prior ADCs or TKIs, and 55.7% having at least three prior lines treatment. Efficacy: A total of 80 T-DXd-naïve patients were enrolled, of whom 75 were evaluable for efficacy. In this population (N=75), JSKN003 demonstrated a confirmed ORR of 54.7% (95% CI: 42.7-66.2). The disease control rate (DCR) and clinical benefit rate (CBR) were 94.7% and 66.7%, respectively. Among 30 patients treated at the RP2D of 6.3 mg/kg, the confirmed ORR was 73.3%, and CBR reached 83.3%. Subgroup analyses by line of therapy showed ORRs of 66.7% in the prior first line group of 15 patients and 63.2% in the prior second line group of 19 patients, respectively. In addition, 8 patients who had previously received T-DXd were enrolled, among whom 7 had evaluable efficacy data. One patient achieved a partial response (PR), four had stable disease (SD), and tumor shrinkage was observed in four patients. These patients were analyzed separately for exploratory purposes. The median duration of response (DoR) in the overall population was 18.4 months (95% CI: 9.9-NE). Median PFS was not mature at the time of data cutoff. The 3-month and 6-month PFS rates were 88.4% (95% CI: 78.8–93.8) and 75.4% (95% CI: 62.3–84.4), respectively. Safety: 15.9% of patients experienced Grade 3 or higher TRAEs. Serious TRAEs were reported in 5.7% of patients. Dose reductions due to TRAEs occurred in 12.5% of patients, and one patient discontinued due to a TRAE. No TRAEs led to death. The most common TRAEs (≥20%) were nausea, increased alanine aminotransferase, decreased white blood cell count, vomiting, anemia, decreased appetite, thrombocytopenia, fatigue, neutropenia, and diarrhea. ILD was reported in 4 patients (4.5%), mostly Grade 1-2; one case was Grade 3. Conclusions JSKN003 demonstrated promising antitumor activity and manageable safety in heavily pretreated HER2-positive breast cancer, including patients previously treated with T-DXd. Its biparatopic HER2 antibody design may enhance target binding and contribute to the observed clinical benefit. A pivotal Phase III trial (JSKN003-301, NCT06846437) is ongoing to compare JSKN003 with T-DM1 in patients with HER2-positive advanced breast cancer who were previously treated with trastuzumab. Title: A pooled analysis of JSKN003, a biparatopic anti-HER2 antibody conjugate (ADC), in patients with advanced HER2-overexpressing (IHC 3+) gastrointestinal tumorsAbstract Number for Publication: 3022Session Type and Title: Poster Session - Developmental Therapeutics - Molecularly Targeted Agents and Tumor BiologySession Date and Time: 6/2/2025 1:30 PM-4:30 PM CDTPresenter: Dan Liu, Beijing Cancer Hospital Methods The pooled analysis was performed to evaluate the efficacy and safety of JSKN003 in HER2-overexpressing (IHC 3+) metastatic gastric cancer or gastroesophageal junction cancer (GC/GEJC) and colorectal cancer (CRC) patients from the Phase I clinical trial (JSKN003-101, NCT05494918) in Australia and the Phase I/II clinical trial (JSKN003-102, NCT05744427) in China. Results As of February 28, 2025, a total of 50 patients with HER2-overexpressing gastrointestinal tumors (27 patients in GC/GEJC and 23 patients in CRC) were enrolled and treated with JSKN003 monotherapy across 7 dose levels: 1 patient at the dose of 2.1 mg/kg, 1 patient at the dose of 4.2 mg/kg, 1 patient at the dose of 5.2 mg/kg, 43 patients at the dose of 6.3 mg/kg, 1 patient at the dose of 7.3 mg/kg, 2 patients at the dose of 8.4 mg/kg and 1 patient at the dose of 10.5 mg/kg. The median age was 60 years (range: 52-66), with 86.0% ECOG PS 1. Most patients were heavily pretreated: 38.0% had at least three lines of prior therapies, 68.0% received anti-HER2 therapy, 48.0% received Irinotecan. Efficacy: Among 48 patients who had at least one tumor assessment after baseline, JSKN003 demonstrated the ORR of 62.5% and the DCR was 93.8%. Among 27 patients with GC/GEJC, the ORR was 63.0% and DCR reached 92.6%. Among 21 patients with CRC, the ORR was 61.9% and DCR reached 95.2%. Among twenty patients with BRAF V600E-wild type, the ORR was 65.0%. Additionally, among 24 patients (4 patients in GC/GEJC and 20 patients in CRC) who were pretreated with irinotecan, the ORR achieved 58.3%. The median DoR in GC/GEJC patients was 9.6 months (95% CI: 3.0-NE), while the median DoR in CRC patients was 12.1 months (95% CI: 5.8-NE). Median PFS was 9.6 months (95%CI: 4.3, 11.6) with 70.4% PFS rate at 6 months in GC/GEJC patients. Median PFS was 13.8 months (95% CI: 6.8, NE) with 88.9% PFS rate at 6 months in CRC patients. Safety: 18.0% of patients experienced Grade 3 or higher TRAEs. Serious TRAEs were reported in 6.0% of patients. Dose reduction due to TRAEs occurred in 20.0% of patients and 16.3% at RP2D. No TEAEs led to discontinuation or death. The most common TRAEs (≥20%) were nausea, diarrhea, decreased appetite, decreased white blood cell count, anemia, fatigue, decreased neutrophil count, decreased platelet count and vomiting. ILD was reported in 3 patients (6.0%), with Grade 1 in 2 patients and Grade 2 in 1 patient. Conclusions JSKN003 demonstrated promising efficacy in heavily pretreated HER2-overexpressing (IHC3+) gastrointestinal tumors including patients previously treated with irinotecan, with a manageable and predictable safety profile. The HER2 biparatopic ADC design may contribute to the observed clinical benefit. About JSKN003 JSKN003 is a bispecific ADC developed based on KN026 using Alphamab's proprietary glycan-specific conjugation platform. JSKN003 can bind HER2 on the surface of tumor cells and release topoisomerase I inhibitors (TOPIi) through cellular endocytosis, thereby exerting anti-tumor effects. Compared with its ADC counterparts, JSKN003 demonstrated better serum stability and stronger bystander effect, which effectively expands the therapeutic window. Results of multiple clinical studies at various stages of JSKN003 in China and Australia have demonstrated favorable safety profile, with promising efficacy of JSKN003 in heavily pretreated patients with advanced solid tumors, especially in patients with platinum-resistant ovarian cancer (PROC), HER2-expressing breast cancer (BC), or high HER2-expressing solid tumors. JSKN003 was granted breakthrough therapy designation by CDE. The designation is for the treatment of platinum-resistant recurrent epithelial ovarian cancer, primary peritoneal cancer, or fallopian tube cancer. Three Phase III clinical studies of JSKN003 for the treatment of HER2-low expressing BC, PROC, and HER2-positive BC as well as multiple exploratory Phase II clinical studies are currently undergoing smoothly. In September 2024, the Company entered a licensing agreement with JMT-Bio Technology Co., Ltd. ("JMT-Bio"), a wholly-owned subsidiary of CSPC Pharmaceutical Group Co., Ltd. ("CSPC") (stock code: pursuant to which, JMT-Bio was granted the exclusive license and sublicense rights to develop, sell, offer for sale and commercialize JSKN003, for the treatment of tumor-related indications (the "Field") in mainland China (excluding Hong Kong, Macau or Taiwan) (the "Territory") and become the sole marketing authorization holder for JSKN003 for the Field in the Territory. Alphamab retains the sole right to supply JSKN003. About Alphamab Oncology Alphamab Oncology is an innovative biopharmaceutical company focusing on oncology therapeutics. By leveraging its proprietary core technology platforms including single-domain antibodies, bispecific antibodies, glycan-specific conjugation, linker-payload, dual-payload antibody conjugation, and subcutaneous high concentration formulation for biologics, the Company has established a product portfolio with differentiated innovation and global competitiveness, covering cutting-edge areas such as antibody-drug conjugates (ADCs), bispecific antibodies, and single-domain antibodies. The Company has one product approved for marketing (Envafolimab, the world's first subcutaneously injectable PD-(L)1 inhibitor), which has made a significant breakthrough in the convenience and accessibility of cancer treatment. Additionally, the Company has multiple bispecific antibodies and bispecific ADCs in clinical stage, while rapidly advancing the preclinical pipeline prioritizing bispecific ADCs and dual-payload ADCs. Multiple strategic collaborations based on innovative products or technology platforms have been established with partners such as CSPC, ArriVent, and Glenmark. Our overarching mission is to make cancer manageable and curable by addressing unmet clinical needs in oncology. Alphamab Oncology is continuously dedicated to the development of effective, safe, and globally competitive anti-tumor drugs, delivering China-innovated cancer therapies to benefit patients worldwide. View original content: SOURCE Alphamab Oncology Sign in to access your portfolio
Associated Press
16-04-2025
- Business
- Associated Press
Sonnet Releases Virtual Investor "What This Means" Segment
- Raghu Rao, Interim Chief Executive Officer and Dr. Richard Kenney, Chief Medical Officer, of Sonnet discuss the recently announced safety data related to SON-1010 - Watch the 'What This Means' video here PRINCETON, N.J., April 16, 2025 (GLOBE NEWSWIRE) -- Sonnet BioTherapeutics Holdings, Inc. (NASDAQ:SONN) (the 'Company' or 'Sonnet'), a clinical-stage company developing immunotherapeutic drugs targeted to the tumor microenvironment (TME), today announced that members of the Sonnet management team participated in a Virtual Investor 'What This Means' segment. As part of the segment, Raghu Rao, Interim Chief Executive Officer and Dr. Richard Kenney, Chief Medical Officer, discussed the recently announced positive safety results of SON-1010 (IL12-FHAB) at the highest dose combined with atezolizumab being evaluated in the Phase 1b/2a clinical trial in adult patients with advanced solid tumors or platinum-resistant ovarian cancer (PROC) (the SB221 study). Mr. Rao and Dr. Kenney also provided insight into next steps for the program. The Virtual Investor 'What This Means' segment featuring Sonnet is now available here. About Sonnet BioTherapeutics Holdings, Inc. Sonnet is an oncology-focused biotechnology company with a proprietary platform for developing targeted biologic drugs with single or bifunctional action. Known as FHAB (Fully Human Albumin-Binding), the technology utilizes a fully human single chain antibody fragment (scFv) that binds to and 'hitch-hikes' on human serum albumin (HSA) for transport to target tissues. Sonnet's FHAB was designed to specifically target tumor and lymphatic tissue, with an improved therapeutic window for optimizing the safety and efficacy of immune modulating biologic drugs. FHAB platform is the foundation of a modular, plug-and-play construct for potentiating a range of large molecule therapeutic classes, including cytokines, peptides, antibodies and vaccines. Sonnet's lead program, SON-1010, or IL-12-FHAB, is in development for the treatment of solid tumors, certain types of sarcoma, and ovarian cancer. SON-1010 is being evaluated in an ongoing Phase 1/2a study through a Master Clinical Trial and Supply Agreement, along with ancillary Quality and Safety Agreements, with Roche in combination with atezolizumab (Tecentriq®) for the treatment of platinum-resistant ovarian cancer (PROC) (NCT05756907). The Company is also evaluating its second program using this platform, SON-1210, an IL12-FHAB-IL15 for solid tumors, in collaboration with the Sarcoma Oncology Center to commence an investigator-initiated and funded Phase 1/2a study for the treatment of pancreatic cancer. Forward-Looking Statements This press release contains certain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934 and Private Securities Litigation Reform Act, as amended, including those relating to the outcome of the Company's clinical trials, the Company's cash runway, the Company's product development, clinical and regulatory timelines, market opportunity, competitive position, possible or assumed future results of operations, business strategies, potential growth opportunities and other statements that are predictive in nature. These forward-looking statements are based on current expectations, estimates, forecasts and projections about the industry and markets in which we operate and management's current beliefs and assumptions. These statements may be identified by the use of forward-looking expressions, including, but not limited to, 'expect,' 'anticipate,' 'intend,' 'plan,' 'believe,' 'estimate,' 'potential,' 'predict,' 'project,' 'should,' 'would' and similar expressions and the negatives of those terms. These statements relate to future events or our financial performance and involve known and unknown risks, uncertainties, and other factors which may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Such factors include those set forth in the Company's filings with the Securities and Exchange Commission. Prospective investors are cautioned not to place undue reliance on such forward-looking statements, which speak only as of the date of this press release. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise. Investor Relations Contact: JTC Team, LLC Jenene Thomas 908-824-0775 [email protected]
Forbes
10-04-2025
- Business
- Forbes
Spin-Off Research Slashes Mural Oncology Valuation
World Cancer Day Stories Template Flat Design Health care Illustration Editable of Square Background ... More for Social media, Greetings Card or Web Ads Mural Oncology (NASDAQ: MURA, $1.12, Market Capitalization $19.30 million), a clinical-stage oncology company focused on discovering and developing immunotherapies, provided an update on the Phase 3 ARTISTRY-7 trial of Nemvaleukin in combination with KEYTRUDA® (pembrolizumab) in patients with Platinum-Resistant Ovarian Cancer (PROC). Based on overall survival data observed in the interim analysis, Mural halted the phase 3 ovarian cancer trial involving nemvaleukin alfa. (for more information, visit Given the setback, the market reacted negatively, and the stock nosedived ~71%. However, Nemvaleukin will continue to be evaluated in a potentially registrational phase 2 trial, ARTISTRY-6, cohort 2, in mucosal melanoma. The topline data readout is expected in 2Q25. Moreover, the preliminary data readouts for less-frequent intravenous dosing of nemvaleukin in patients with cutaneous melanoma are expected in 2Q25 for cohort 3 of ARTISTRY-6 (monotherapy) and in 2H25 for cohort 4 of ARTISTRY-6 (combination therapy), subject to patient enrolment. Mural Oncology Price Performance and Spin-Off Details Earlier in 4Q24, the Company expanded its pipeline by nominating two development candidates, one for its interleukin-18 (IL-18) program and another for its IL-12 program. MURA-8518 is designed to be a half life extended, binding protein-resistant IL-18 in order to overcome the native cytokine's limitations as a therapeutic. Mural expects to submit an Investigational New Drug (IND) Application or Clinical Trial Application (CTA) for a phase 1 trial of MURA-8518 in 4Q25. MURA-7012 is comprised of targeted split IL-12 sub-units that preferentially self-assemble at the tumor site and are designed to limit systemic exposure. Key Data MURA and Top 5 Shareholders Valuation and RecommendationOur fair value estimate for Mural Oncology is $2.75 per share (Previously: $12.00 per share), based on a terminal growth rate of 2% and WACC of 14.01%. The downgrade in our target price factors in the recent announcement of the termination of the Phase 3 trial of ARTISTRY-7. The trial investigated the efficacy of nemvaleukin alfa with Merck & Co., Inc.'s (MRK) Keytruda against chemotherapy for the treatment of platinumresistant ovarian cancer (PROC). The company stated that the combination therapy failed to demonstrate a statistically significant improvement in overall survival (OS) (the primary endpoint) compared to the standard of care, due to which the company's potential for revenue has declined. Due to this, the stock took a significant hit and has fallen by ~71% since the announcement. Due to the termination of ARTISTRY-7 for ovarian cancer, the company now looks forward to a top-line data readout for ARTISTRY-6, cohort 2 in mucosal melanoma in 2Q25. Along with that, Nemvaleukin is also set for preliminary data readouts in 2Q25 for less-frequent intravenous dosing in ARTISTRY-6 cohort 3 and 2H25 for ARTISTRY 6 cohort 4, both targeting cutaneous melanoma. Mural also expanded its pipeline in 4Q24 by nominating two development candidates, named MURA-8518 and MURA- 7012. As of FY24, Mural Oncology has spent ~$607 million from FY21 to FY24, which implies the company is heavily investing in its pipeline drug, Nemvaleukin. Furthermore, the company stated that the current cash, cash equivalents, and marketable securities of $144.4 million are expected to fund its operations into 1Q26, and the company has not provided any updates on the new cash injection. As the bulk of cash would be used for operational purposes, we have assumed a net cash of $14 million (~10% of cash) in our DCF valuation model. In view of its high-risk business model and no further indication of cash injection or plans to raise capital, the stock offers an asymmetric binary payout depending on the success of ongoing trials on Nemvaleukin candidates for melanoma therapy. We maintain our 'Long Term High Risk Buy' rating on Mural Oncology Plc with an implied upside of 145.4% from the current market price of $1.12 as of 4/9. About the ARTISTRY-7 Trial ARTISTRY-7 was a phase 3 trial comparing nemvaleukin in combination with pembrolizumab vs. investigator choice single-agent chemotherapy in patients with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer. This trial completed enrollment with 456 patients, and its primary endpoint was overall survival (OS). In the interim analysis of ARTISTRY-7, the safety profile was generally consistent with previously reported data. However, the Company noted that in the prespecified interim analysis conducted by the independent data monitoring committee, nemvaleukin in combination with pembrolizumab did not achieve a statistically significant improvement in overall survival versus the investigator's choice of chemotherapy alone, and the company believed the study was highly unlikely to achieve success at the final analysis. Median overall survival was 10.1 months for patients treated with nemvaleukin in combination with pembrolizumab and 9.8 months for patients treated with investigator's choice chemotherapy (hazard ratio: 0.98). Next Steps & Milestones: Despite the ARTISTRY-7 trial setback, Mural Oncology has set several key milestones in its development trajectory over the next year.2Q25: Top-line data readout of Cohort 2 of ARTISTRY-6, Mural's potentially registrational phase 2 trial of nemvaleukin monotherapy in patients with unresectable or metastatic mucosal melanoma previously treated with immune checkpoint blockade. Nemvaleukin has been granted an Orphan Drug Designation by the FDA for the treatment of mucosal melanoma.1H25: Preliminary data readout of Cohort 3 of ARTISTRY-6, an evaluation of a less frequent intravenous (LFIV) dose of nemvaleukin monotherapy in patients with cutaneous melanoma. The company is conducting a trial to evaluate the activity and further characterize the safety of nemvaleukin with LFIV dosing in patients with cutaneous melanoma. 2H25: Preliminary data readout of Cohort 4 of ARTISTRY-6, an evaluation of an LFIV dose of nemvaleukin in combination with pembrolizumab in patients with cutaneous melanoma.1H26: Submission of Investigational New Drug or Clinical Trial Application for a phase 1 trial of MURA-8518, its IL-18 development candidate. These milestones reflect Mural's commitment to addressing unmet medical needs in oncology and advancing innovative immuno-oncology therapies. Financial & Market Outlook: • Mural Oncology is positioned to address large unmet medical needs in mucosal and cutaneous melanoma, which has limited therapeutic options. According to Bloomberg Intelligence report, the global market for melanoma drugs can reach nearly $11 billion in 2027 from $8 billion in 2023, and without risk adjustment for pipeline products and key line extensions, sales are projected to pass $12 billion by 2030. The company expects nemvaleukin to be a key asset for driving growth. • With an adequate cash runway into 1Q26, the company is well-positioned to continue its clinical development programs. At the end of FY24, the Company reported a cash balance is $144.4 million. 4Q24 & FY24 Results Review 4Q24 On 3/11, the company released its 4Q and FY24 results. For 4Q24, Research and Development (R&D) expenses were $28.7 million in 4Q24 compared to $42.2 million in 4Q23. The decrease was primarily due to lower employee-related expenses, including a non-cash share-based employee compensation charge in 4Q23, as a result of the impact of the modification of share-based awards in connection with the separation from Alkermes. Moreover, the timing of patient enrolment in the ARTISTRY-7 trial and the winding down of the ARTISTRY-1 and ARTISTRY-2 trials during 2024 also contributed to the lower R&D expenses in 4Q24. General and Administrative (G&A) expenses were $7.2 million in 4Q24 compared to $16.2 million in 4Q23. The decline in G&A expense was attributable to a decreased employee-related expenses compared to those previously allocated to Mural Oncology by Alkermes prior to the separation and to one time increases in employee-related expenses in 2023, including a non-cash share-based employee compensation charge in 4Q23 as a result of the impact of the modification of share based-awards in connection with the separation. Ultimately, the Company's net loss declined to $34.3 million in 4Q24, compared to $59.5 million in 4Q23, which included the one-time charges of ~$11.7 million related to the separation from Alkermes and conversion of Alkermes employee equity awards into Mural equity. Although loss per share improved to $2.01 in 4Q24 compared to $3.57 in 4Q23, it was a miss as compared to the consensus estimate of $2.10 per share. FY24 Research and Development (R&D) expenses were $110.7 million in FY24 compared to $165.5 million in the prior-year period. The decrease was primarily due to lower employee-related expenses compared to the personnel previously allocated to Mural by Alkermes prior to the separation. The decline in R&D expenses was due to decreased spending on the ARTISTRY-1 and ARTISTRY-2 trials as activities related to these trials wound down in 2023 and lower manufacturing spending on other programs. General and Administrative (G&A) expenses were $27.6 million in FY24 compared to $30.7 million in the prior-year period. The decrease in G&A expense was primarily attributable to lower employee-related expenses, partially offset by costs associated with operating as a standalone company after the Separation. Ultimately, the Company's net loss declined to $128.5 million in FY24, compared to $207.4 million in FY23, which included one-time charges related to the separation from Alkermes and the conversion of Alkermes employee equity awards into Mural equity. Consequently, loss per share improved to $7.58 in FY24 compared to $12.43 in FY23. The cash balance at the end of FY24 was ~$144.4 million. Valuation In recent developments, MURA terminated the Phase 3 trial of ARTISTRY-7, the Nemvaleukin candidate for ovarian cancer. We have adjusted our model to factor in this development, and our revenue model is driven by Nemvaleukin's ARTISTRY-6 cohort 2, cohort 3 and cohort 4 candidates, currently in potential registration, for the monotherapy of patients with mucosal & cutaneous melanoma. Mural expects topline readouts for ARTISTRY-6 cohort 2 in 2Q25 and preliminary data read-outs for ARTISTRY-6 cohort 3 and cohort 4 candidates for cutaneous melanoma in 2Q and 2H25. Mural also expanded its pipeline in 4Q24 by nominating two development candidates, named MURA-8518 and MURA-7012, designed to deliver a more sustained immune response and leverage native IL-12's anti-tumor potency while mitigating its hallmark toxicity. With the ovarian cancer terminated, the company now majorly focuses on melanoma therapy. According to Bloomberg, the market for melanoma drugs can reach nearly $11 billion in 2027 from $8 billion in 2023. We estimate Nemvaleukin to achieve ~$40.2 million in net revenue by 2032 with ~0.5% market share. Intrinsic Valuation - Mural Oncology DCF Valuation - Using a discounted cash flow methodology, our fair value estimate for Mural Oncology stands at $2.75 (Previously: $12.00) per share. The downgrade in our target price factors in the recent announcement of the termination of the Phase 3 trial of ARTISTRY-7. Our DCF thesis assumes a terminal growth rate of 2%, WACC of 14.01% and a share count of 17.2 million. The company stated that the current cash, cash equivalents, and marketable securities of $144.4 million are expected to fund its operations into 1Q26, and the company has not provided any outlook on whether further cash will be injected. As the bulk of cash would be used for operational purposes, we have assumed a net cash of $14 million (~10% of cash) in our DCF valuation model. Our implied equity value for Mural Oncology stands at $47 million, which is mostly due to the present value of our estimated terminal cash flow at $26 to Target Price/Valuation - For Mural Oncology, clinical outcomes from clinical trials, especially for products such as nemvaleukin, could impact how investors view its outlook. Company DescriptionMural Oncology Mural Oncology is leveraging its novel protein engineering platform to develop cytokine-based immunotherapies for cancer treatment. By combining its expertise in cytokine biology, immune cell modulation, and protein engineering platforms, MURA is developing medicines to deliver meaningful and clinical benefits to people with cancer. The company's mission is to broaden the potential and reach of cytokine-based immunotherapies to improve the lives of patients. MURA's lead candidate, nemvaleukin, is currently in potentially registrational trials in mucosal and cutaneous melanoma. Mural Oncology has its registered office in Dublin, Ireland, and its primary facilities in Waltham, Mass.
Yahoo
17-03-2025
- Business
- Yahoo
Investigational Rinatabart Sesutecan (Rina-S®) Continues to Show Encouraging Antitumor Activity in Patients with Advanced Ovarian Cancer
Media ReleaseCOPENHAGEN, Denmark; March 17, 2025 Results from Phase 2 RAINFOLTM-01 trial (B1 cohort) showed that with a median on-study follow-up of 48 weeks, Rina-S 120 mg/m2 led to a confirmed objective response rate (ORR) of 55.6% and median duration of response (mDOR) was not reached Phase 2 RAINFOLTM-01 and Phase 3 RAINFOLTM-02 trials evaluating the safety and efficacy of Rina-S at 120 mg/m2 in patients with platinum resistant ovarian cancer (PROC) are actively recruiting Genmab A/S (Nasdaq: GMAB) announced today updated data from cohort B1 of the Phase 1/2 RAINFOL-01 study of rinatabart sesutecan (Rina-S®), an investigational folate receptor-alpha (FRα)-targeted, TOPO1 antibody-drug conjugate (ADC) that showed Rina-S 120 mg/m2 every 3 weeks (Q3W) resulted in a confirmed objective response rate (ORR) of 55.6% (95% CI: 30.8-78.5) in heavily pre-treated ovarian cancer (OC) patients regardless of FRα expression levels. With a median on-study follow-up of 48 weeks, 1 out of 10 patients experienced disease progression and the median duration of response (mDOR) was not reached (95% CI: 40.14-NR). The data are from the dose expansion cohort of the multi-part study evaluating the safety and efficacy of Rina-S as a single agent in solid tumors that are known to express FRα and were presented at the 2025 Society of Gynecologic Oncology Annual Meeting on Women's Cancer® (SGO) in Seattle, Washington. 'The antitumor activity observed in the dose expansion cohort continues to demonstrate the potential for a much-needed treatment option for patients with PROC, who have historically had poor prognosis. I am hopeful that further exploration of Rina-S will lead to advancements in the treatment landscape.' said Elizabeth Lee, M.D., a medical oncologist in the gynecologic oncology program at Dana-Farber. The B1 cohort is a dose expansion study in patients with histologically or cytologically confirmed advanced OC (epithelial ovarian cancer, primary peritoneal cancer or fallopian tube cancer). Rina-S 120 mg/m2 Q3W at a median on-study follow-up of 48 weeks showed encouraging antitumor activity; the confirmed ORR was 55.6% (95% CI: 30.8-78.5), the disease control rate (DCR) was 88.9% (95% CI: 65.3-98.6), and the median duration of response (mDOR) was not reached (95% CI: 40.14-NR). In the 18 patients evaluable for response treated with 120 mg/m2 Q3W, complete responses were observed in 4 patients (2 confirmed; 2 unconfirmed) and 8 patients experienced confirmed partial responses (44.4%). Most responses with Rina-S 120 mg/m2 were observed early (at week 6). Only one patient in the 120 mg/m2 treatment arm was not evaluable. In the Rina-S 100 mg/m2 Q3W treatment arm (N=22), at a median on study follow-up of 46 weeks, the confirmed ORR was 22.7% (95% CI: 7.8-45.4), the DCR was 86.4% (95% CI: 65.1-97.1), and the mDOR was not reached (95% CI, 16.3-NR). Partial responses were observed in 4 patients (18.2%) and 1 patient (4.5%) experienced a complete response. Rina-S 120 mg/m2 has been selected for further evaluation in the RAINFOL-01 and Phase 3 RAINFOL-02 trials for patients with platinum resistant ovarian cancer (PROC). In this Phase 1/2 study, common treatment-emergent adverse events (TEAEs) included anemia, nausea, neutropenia, leukopenia, fatigue, thrombocytopenia, vomiting, diarrhea, alopecia, and hypokalemia. Dose reductions and treatment discontinuations were infrequent and no new safety signals were observed. 'The updated results reinforce the potential of Rina-S and further validate our development approach in advanced ovarian cancer,' said Judith Klimovsky, M.D., Executive Vice President and Chief Development Officer of Genmab. 'We are excited to keep moving forward with the ongoing Phase 3 trial, to evaluate the potential of Rina-S as a treatment option for patients facing this challenging disease.' The safety and efficacy of rinatabart sesutecan has not been established for these investigational uses. About the RAINFOLTM -01 Trial RAINFOL-01 (NCT05579366) is an open-label, multicenter Phase 1/2 study, designed to evaluate the safety and efficacy of rinatabart sesutecan (Rina-S) as a single agent Q3W at various doses in solid tumors that are known to express FRα. The study consists of multiple parts including Part A dose-escalation cohorts; Part B tumor-specific monotherapy dose-expansion cohorts; Part C platinum-resistant ovarian cancer (PROC) cohort; and Part D combination therapy cohorts. Part B of the trial includes the B1 cohort, a dose expansion study in patients with histologically or cytologically confirmed advanced OC (epithelial ovarian cancer, primary peritoneal cancer, or fallopian tube cancer). Patients were randomized 1:1 to 100 mg/m2 and 120 mg/m2 dose cohorts. Median age was 62.5 and 64.5 years in the 100 mg/m2 and 120 mg/m2 cohorts, respectively. Study participants were previously treated with a median of 3 prior lines of therapy (range 1-4). Patients received prior treatment with bevacizumab (90.9% in the 100 mg/m2 group and 90.0% in the 120 mg/m2 group respectively), PARP inhibitors (68.2%; 65%), and mirvetuximab soravtansin (18.2%; 19%). Initial results from Part B of this trial were presented during a mini-oral session at the European Society of Medical Oncology Congress 2024 (ESMO). About Ovarian CancerOvarian cancer is a major global health issue, with over 320,000 new cases diagnosed annually worldwide.i It ranks as the eighth most common cancer and the eighth leading cause of cancer-related deaths among women The disease is often diagnosed at an advanced stage due to its subtle and non-specific symptoms, such as abdominal bloating, pelvic pain and difficulty Standard of care for platinum resistant ovarian cancer typically involves single agent chemotherapy (pegylated liposomal doxorubicin (PLD), topotecan, gemcitabine or paclitaxel).iv Approximately 70-90% of women with advanced-stage ovarian cancer worldwide experience a recurrence after initial treatment.v Ovarian cancer has a low five-year survival rate, which varies significantly by region, but generally hovers around 30-50%.vi,vii About Rinatabart Sesutecan (Rina-S®; GEN1184)Rinatabart sesutecan (Rina-S; GEN1184) is a FRα-targeted, TOPO1 ADC, currently being evaluated for the potential treatment of ovarian cancer and other FRα-expressing cancers. A Phase 3 trial (RAINFOL-02, NCT06619236) evaluating Rina-S in patients with platinum resistant ovarian cancer compared to treatment of investigator's choice is ongoing. In January 2024, the U.S. Food and Drug Administration granted Fast Track designation to Rina-S for the treatment of patients with FRα-expressing high-grade serous or endometrioid platinum-resistant ovarian cancer. Please visit for more information. About Genmab Genmab is an international biotechnology company with a core purpose of guiding its unstoppable team to strive toward improving the lives of patients with innovative and differentiated antibody therapeutics. For more than 25 years, its passionate, innovative and collaborative team has invented next-generation antibody technology platforms and leveraged translational, quantitative and data sciences, resulting in a proprietary pipeline including bispecific T-cell engagers, antibody-drug conjugates, next-generation immune checkpoint modulators and effector function-enhanced antibodies. By 2030, Genmab's vision is to transform the lives of people with cancer and other serious diseases with knock-your-socks-off (KYSO) antibody medicines®. Established in 1999, Genmab is headquartered in Copenhagen, Denmark, with international presence across North America, Europe and Asia Pacific. For more information, please visit and follow us on LinkedIn and X. Contact: David Freundel, Senior Director, Global Communications & Corporate AffairsT: +1 609 430 2481; E: dafr@ Andrew Carlsen, Vice President, Head of Investor RelationsT: +45 3377 9558; E: acn@ Media Release contains forward looking statements. The words 'believe,' 'expect,' 'anticipate,' 'intend' and 'plan' and similar expressions identify forward looking statements. Actual results or performance may differ materially from any future results or performance expressed or implied by such statements. The important factors that could cause our actual results or performance to differ materially include, among others, risks associated with preclinical and clinical development of products, uncertainties related to the outcome and conduct of clinical trials including unforeseen safety issues, uncertainties related to product manufacturing, the lack of market acceptance of our products, our inability to manage growth, the competitive environment in relation to our business area and markets, our inability to attract and retain suitably qualified personnel, the unenforceability or lack of protection of our patents and proprietary rights, our relationships with affiliated entities, changes and developments in technology which may render our products or technologies obsolete, and other factors. For a further discussion of these risks, please refer to the risk management sections in Genmab's most recent financial reports, which are available on and the risk factors included in Genmab's most recent Annual Report on Form 20-F and other filings with the U.S. Securities and Exchange Commission (SEC), which are available at Genmab does not undertake any obligation to update or revise forward looking statements in this Media Release nor to confirm such statements to reflect subsequent events or circumstances after the date made or in relation to actual results, unless required by law. Genmab A/S and/or its subsidiaries own the following trademarks: Genmab®; the Y-shaped Genmab logo®; Genmab in combination with the Y-shaped Genmab logo®; HuMax®; DuoBody®; HexaBody®; DuoHexaBody®, HexElect®, Rina-S®and KYSO®. i World Cancer Research Fund International. Accessed August World Ovarian Cancer Coalition. Accessed August Dilley, James et al. Ovarian cancer symptoms, routes to diagnosis and survival - Population cohort study in the 'no screen' arm of the UK Collaborative Trial of Ovarian Cancer Screening (UKCTOCS). Gynecologic oncology vol. 158,2 (2020): 316-322. doi:10.1016/ iv Eskander RN, Moore KN, Monk BJ, Herzog TJ, Annunziata CM, O'Malley DM and Coleman RL (2023) Overcoming the challenges of drug development in platinum-resistant ovarian cancer. Front. Oncol. 13:1258228Ovarian Cancer Research Alliance. Ovarian Cancer Research Alliance. European Institute of Women's Health. Accessed August American Cancer Society. Stages of Ovarian Cancer. Accessed August 2024. Media Release no. i05CVR no. 2102 3884LEI Code 529900MTJPDPE4MHJ122 Genmab A/SCarl Jacobsens Vej 302500 ValbyDenmark Attachment 170325 MRi05 Rina-S SGO DataSign in to access your portfolio
Associated Press
16-03-2025
- Business
- Associated Press
Sutro Biopharma Presents Data from Dose-Optimization Portion of REFRαME-O1 Trial in Patients with Platinum Resistant Ovarian Cancer at SGO 2025
SOUTH SAN FRANCISCO, Calif., March 15, 2025 (GLOBE NEWSWIRE) -- Sutro Biopharma, Inc. (Sutro or the Company) (NASDAQ: STRO), an oncology company pioneering site-specific and novel-format antibody drug conjugates (ADCs), today announced expanded data in a late-breaking oral presentation from the dose-optimization portion of the REFRαME-O1 trial with luveltamab tazevibulin (luvelta) in patients with platinum resistant ovarian cancer (PROC) at the Society of Gynecologic Oncology (SGO) Annual Meeting on Women's Cancer®. The SGO Annual Meeting will take place from March 14-17, 2025 in Seattle, Washington. In this study, luvelta demonstrated encouraging antitumor activity in patients with late-stage ovarian cancer across all levels of Folate Receptor-α (FRα) expression of 25% or greater, including an improved overall response rate (ORR), a low discontinuation rate, and a consistent safety profile across dose levels. Based on these findings, Sutro selected the optimized dose of luvelta: 5.2 mg/kg + G-CSF for two cycles then continued on 4.3 mg/kg. 'These data demonstrate the potential for improved patient responses compared to standard chemotherapy in PROC, especially patients whose FRα expression falls within the range of at least 25% to less than 75% 2+, which remains an important unmet medical need,' commented Dr. Jung Yun Lee, Professor, Gynecologic Oncologist, Yonsei Cancer Center and Severance Hospital, Yonsei University College of Medicine, Seoul, Republic of Korea. Late-Breaking Oral Presentation Highlights: At the selected optimized dose (5.2 mg/kg), luvelta achieved an ORR of 32%1 and a disease control rate (DCR) of 96% compared to an ORR of 13.8% and a DCR of 69% for the 4.3 mg/kg group. The demonstrated clinical activity in the 5.2 mg/kg group was consistent in patients across all levels of FRα expression of 25% or greater, with an ORR of 30.8% and a DCR of 100% for positive staining (PS) 2+ ≥75% (eligible for approved FRα-targeting ADC) and an ORR of 33.3%1 and DCR of 91.7%1 for PS2+ < 75%. Safety was consistent across dosing groups, with no new safety signals observed and neutropenia well-managed. The majority of patients across both dose cohorts received prior bevacizumab. The presentation will be accessible through the News & Events page of the Investor Relations section of the company's website at The Company recently announced that it is deprioritizing investment into the development of luvelta across all indications. Sutro continues to explore out-licensing opportunities to deliver the promise of luvelta's benefit to patients with unmet need in platinum resistant ovarian cancer and beyond. 1 Immediately a fter data extraction, one additional patient experienced a confirmed PR and is included in the analysis. About Luveltamab Tazevibulin Luveltamab tazevibulin, abbreviated as 'luvelta' and formerly known as STRO-002, is a FRα-targeting antibody-drug conjugate (ADC) designed to treat a broad range of patients with ovarian cancer, including those with lower FRα-expression who are not eligible for approved treatment options targeting FRα. Developed and manufactured with Sutro's cell-free XpressCF® platform, luvelta is a homogeneous ADC with four hemiasterlin cytotoxins per antibody, precisely positioned to efficiently deliver to the tumor while ensuring systemic stability after dosing. The U.S. Food and Drug Administration (FDA) has granted luvelta a Fast Track designation for Ovarian Cancer, as well as Orphan and Rare Pediatric Disease designations for CBF/GLIS2 Pediatric AML. About Sutro Biopharma Sutro Biopharma, Inc., is relentlessly focused on the discovery and development of precisely designed cancer therapeutics to transform what science can do for patients. Sutro's fit-for-purpose technology, including cell-free XpressCF®, provides the opportunity for broader patient benefit and an improved patient experience. Sutro is advancing a robust early-stage pipeline of novel exatecan and dual-payload antibody drug conjugates (ADCs), coupled with high-value collaborations and industry partnerships, which validate its continuous product innovation. Sutro is headquartered in South San Francisco. For more information, follow Sutro on social media @Sutrobio, or visit Forward-Looking Statements This press release contains forward-looking statements within the meaning of the 'safe harbor' provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, anticipated preclinical and clinical development activities, including enrollment and site activation; timing of announcements of clinical results, trial initiation, and regulatory filings; outcome of discussions with regulatory authorities; potential benefits of luvelta and the Company's other product candidates and platform; potential business development and partnering transactions; potential market opportunities for luvelta and the Company's other product candidates. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. Although the Company believes that the expectations reflected in such forward-looking statements are reasonable, the Company cannot guarantee future events, results, actions, levels of activity, performance or achievements, and the timing and results of biotechnology development and potential regulatory approval is inherently uncertain. Forward-looking statements are subject to risks and uncertainties that may cause the Company's actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the Company's ability to advance its product candidates, the receipt and timing of potential regulatory designations, approvals and commercialization of product candidates, the market size for the Company's product candidates to be smaller than anticipated, clinical trial sites, supply chain and manufacturing facilities, the Company's ability to maintain and recognize the benefits of certain designations received by product candidates, the timing and results of preclinical and clinical trials, the Company's ability to fund development activities and achieve development goals, the Company's ability to protect intellectual property, and the Company's commercial collaborations with third parties and other risks and uncertainties described under the heading 'Risk Factors' in documents the Company files from time to time with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and the Company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof. Investor Contact Emily White Sutro Biopharma (650) 823-7681 Media Contact Amy Bonanno Lyra Strategic Advisory
