Core Insights - Rhythm Pharmaceuticals presented new data on acquired hypothalamic obesity at the Endocrine Society's Annual Meeting (ENDO 2025), highlighting the efficacy of setmelanotide and bivamelagon in reducing body mass index (BMI) in affected patients [1][2][3]. Group 1: Clinical Data and Efficacy - The Phase 2 SIGNAL trial demonstrated that bivamelagon, a daily oral MC4R agonist, achieved statistically significant and clinically meaningful reductions in BMI over 14 weeks [2]. - The Phase 3 TRANSCEND trial showed setmelanotide resulted in a -9.3% BMI reduction in the 600mg cohort, -7.7% in the 400mg cohort, and -2.7% in the 200mg cohort, with a placebo cohort showing a 2.2% increase in BMI [4][5]. - A placebo-adjusted difference of -19.8% in BMI reduction was observed across 120 participants, with significant reductions noted in various age and sex subgroups [5][6]. Group 2: Patient Impact and Future Potential - The data from the trials suggest that both bivamelagon and setmelanotide could become transformative therapeutic options for patients with acquired hypothalamic obesity, potentially establishing a new standard of care [2][4]. - Rhythm estimates that there are between 5,000 to 10,000 individuals living with hypothalamic obesity in the U.S., indicating a significant patient population that could benefit from these treatments [11]. Group 3: Company Overview - Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases, with its lead asset, setmelanotide, approved for treating obesity related to specific genetic conditions [9][12]. - The company is advancing a broad clinical development program for setmelanotide and other investigational MC4R agonists, indicating a commitment to expanding treatment options for patients with rare diseases [9].

Core Insights - Rhythm Pharmaceuticals presented new data on acquired hypothalamic obesity at the Endocrine Society's Annual Meeting (ENDO 2025), highlighting the efficacy of setmelanotide and bivamelagon in reducing body mass index (BMI) in affected patients [1][2][3] Group 1: Clinical Data and Efficacy - The Phase 3 TRANSCEND trial demonstrated significant BMI reductions with setmelanotide, showing a -9.3% reduction in the 600mg cohort, -7.7% in the 400mg cohort, and -2.7% in the 200mg cohort, while the placebo cohort experienced a 2.2% increase [4][5] - A placebo-adjusted difference of -19.8% in BMI reduction was observed across 120 participants, with significant reductions noted in various age and sex subgroups [5][6] - Bivamelagon, a daily oral MC4R agonist, also showed statistically significant BMI reductions in a Phase 2 trial, indicating its potential as a transformative treatment option for patients with acquired hypothalamic obesity [2][3] Group 2: Patient Population and Market Potential - Acquired hypothalamic obesity is a rare condition affecting an estimated 5,000 to 10,000 individuals in the U.S., with similar prevalence in Japan and the E.U. [11][10] - The condition is characterized by rapid weight gain and hyperphagia due to damage to the hypothalamic region of the brain, often following brain tumors or their treatment [10][11] - The presentations at ENDO 2025 underscore the potential for MC4R-targeted therapies to become the standard of care for this patient community if approved [2][3]

SAN DIEGO, July 09, 2025 (GLOBE NEWSWIRE) -- Calidi Biotherapeutics, Inc. (“Calidi” or the “Company”) (NYSE American: CLDI), a clinical-stage biotechnology company pioneering the development of targeted therapies with the potential to deliver genetic medicines to distal sites of disease, today announced the entry into a definitive agreement for the immediate exercise of certain outstanding Series A, Series B-1, Series C-1, Series D, Series E and Series F Warrants (the “Existing Warrants”) to purchase up to ...

[Applause] All right. So, today I'm going to talk to you about cancer and about three very important words. You have cancer.I remember those words sort of tumbling out of my mouth. They weren't elegantly polished in the way I had sort of learned in medical school, actually here at Duke, sitting in the building across the street. They didn't have any of the the poignant pauses that I had practiced in the mirror on my first day as a doctor at the University of Chicago.None of the elegance, just words. Sort of ...

Core Insights - Hoth Therapeutics' investigational candidate HT-001 achieved a 100% response rate in at least one primary efficacy endpoint in its ongoing Phase 2a clinical study for EGFR inhibitor-induced skin toxicities [2][7] - The company will host a Key Opinion Leader event to discuss the interim results and potential impact of HT-001 on supportive care standards for oncology patients [1] Clinical Trial Highlights - The Phase 2a trial (CLEER-001) demonstrated that 100% of enrolled patients achieved at least one primary endpoint of clinical dermatologic improvement [7][8] - Over 65% of patients reported reductions in pain and pruritus, with 0% requiring dose reduction or discontinuation of their EGFR inhibitor therapy [7][8] - HT-001 is a once-daily topical gel that targets neuroinflammatory pathways without causing immunosuppression or systemic toxicity [4] Mechanism of Action - HT-001 is formulated with an FDA-approved neurokinin-1 receptor antagonist (NK1RA) that mitigates inflammatory pathways triggered by EGFR inhibition, particularly those driven by Substance P [4][5] - Preclinical data showed significant reductions in dermatitis and alopecia severity, as well as inflammatory markers in rat models co-treated with erlotinib [5][9] Regulatory and Development Pathway - HT-001 is being advanced under the 505(b)(2) regulatory pathway, which allows the use of existing safety data to expedite development [10] - The company is currently planning a Phase 2b/3 trial following the ongoing Phase 2a trial [13]

Nuvalent (NUVL) Update / Briefing June 24, 2025 08:00 AM ET Speaker0 Good morning, and welcome to New Zealand's conference call. At this time, all participants are in a listen only mode. There will be a question and answer session after the prepared remarks. Please be advised that this call is being recorded at the company's request. I would now like to turn the call over to Alex Stalcom, CFO of Nuvalent. You may begin. Speaker1 Thank you all for joining us today. Earlier this morning we issued a press rele ...

Aligned with FDA on NDA submission strategy for TKI pre-treated patients with advanced ROS1-positive NSCLC and participation in Real-Time Oncology Review; the company plans to initiate a rolling NDA submission in July 2025 with target completion in the third quarter of 2025 In 117 ROS1 TKI pre-treated patients, including 50% who had received ≥ 2 prior ROS1 TKIs ± chemotherapy, ORR by BICR was 44% (95% CI: 34, 53) with initial estimated durability of response of 78% at the 12-month landmark and 62% at the 1 ...

CAMBRIDGE, Mass., June 18, 2025 /PRNewswire/ -- Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, today announced the appointment of Christy Oliger to its Board of Directors."Christy's proven success in delivering new therapies to patients living with cancer strengthens our Board as we evolve from a development-stage company toward potential commercialization," said James Porter, Ph.D., C ...

Regeneron Pharmaceuticals (REGN) FY 2025 Annual General Meeting June 13, 2025 10:30 AM ET Speaker0 Good morning, and welcome to the twenty twenty five Annual Shareholder Meeting of Regeneron Pharmaceuticals. The meeting will begin after the following message. Speaker1 At Regeneron, we've always pursued bold science to drive our purpose, transforming lives through groundbreaking innovation. That principle is just as strong today as it was when the company was founded. Speaker2 We committed to not just treati ...

An updated edition of the April 16, 2025, article.The global cancer treatment market is transforming rapidly, driven by an increasing demand for more effective, less toxic therapies. As per the American Cancer Society, the United States alone is expected to see 2,041,910 new cancer cases and 618,120 cancer-related deaths in 2025. While advances in early detection and treatment have contributed to a decline in mortality rates for certain cancers, the overall rise in cancer incidence continues to push global ...