Rhythm Pharmaceuticals (RYTM) Update / Briefing July 09, 2025 08:00 AM ET Speaker0 Good day and thank you for standing by. Welcome to the Rhythm Pharmaceuticals Top Line Results from Phase II Trial Evaluating Bivamilagon in Acquired Hypothalamic Obesity Conference Call. At this time, participants are in a listen only mode. After the speakers' presentation, there will be a question and answer session. To ask the questions in the session, you will need to press 11 on your telephone. You will then hear an auto ...

Core Viewpoint - ProKidney Corp. has experienced significant stock volatility following positive Phase 2 clinical trial results for its REGEN-007 study, which shows promise for treating chronic kidney disease (CKD) and diabetes [2][5]. Group 1: Clinical Trial Outcomes - The Phase 2 trial results indicated a 78% improvement in the annual decline of estimated glomerular filtration rate (eGFR) for Group 1 participants who received two rilparencel injections [6]. - Group 2 participants, who received a single injection with a conditional second injection, showed a 50% improvement in annual eGFR decline, although this result was not statistically significant [6]. - These findings represent a significant clinical milestone, as CKD affects millions globally and treatment options are limited [2]. Group 2: Market Response and Stock Performance - Following the positive trial results, ProKidney's stock surged over 500% on July 8, although it remains over 70% below its 2023 peak of approximately $13 [2]. - Current analyst consensus price targets are around $4.00, suggesting the stock may be fairly valued at its current levels [3]. Group 3: Financial Position - ProKidney holds a robust cash balance of $358 million, representing 81% of its total assets, and has a minimal debt load of $3.2 million, resulting in a debt-to-equity ratio below 1% [3]. - The company incurred operating expenditures totaling $185 million over the past year and has no commercial products generating revenue, necessitating continuous observation of its cash burn rate [4]. Group 4: Risks and Challenges - The uncertainty surrounding the Phase 3 trial poses a significant risk, as success in Phase 2 does not guarantee success in subsequent phases or regulatory approvals [5]. - ProKidney faces competitive risks from other companies developing therapies for CKD and has a limited pipeline reliant on rilparencel, which adds to its risk profile [5]. - The stock has shown substantial price fluctuations, with a notable decline of 91% from a high of $13.78 on March 13, 2023, to $1.21 on November 17, 2023 [5].

Clinical Trial Results - Bivamelagon achieved statistically significant BMI reductions in patients with acquired hypothalamic obesity at 14 weeks[5] - At 14 weeks, the 200mg Bivamelagon group saw a mean BMI reduction of 268%, the 400mg group saw a mean BMI reduction of 769%, and the 600mg group saw a mean BMI reduction of 931%[27] - In the placebo group, there was a mean BMI increase of 218% from baseline at 14 weeks[27] - A significant percentage of patients achieved clinically meaningful BMI reductions: 167% in the 200mg Bivamelagon group, 714% in the 400mg group, and 75% in the 600mg group achieved at least a 5% BMI reduction[33] - Bivamelagon also achieved meaningful reductions in 'most' hunger scores at Week 14[44] Safety and Tolerability - Safety and tolerability results were generally consistent with an MC4R MOA and setmelanotide trials in acquired hypothalamic obesity, with limited instances of localized hyperpigmentation[5] - The most common AEs with >=10% in all BIVA dosing (N=21) were nausea, diarrhea, vomiting, and headache[47] Market Opportunity and Future Development - Rhythm Pharmaceuticals intends to request an End-of-Phase 2 meeting with the FDA with intentions to move forward to Phase 3[5] - The company estimates the U S prevalence of acquired hypothalamic obesity to be 5,000 – 10,000 patients[13] - U S patent protection for bivamelagon and RM-718 extends into the 2040s[13]

Phase 1/2 clinical study evaluating seclidemstat in patients with MDS and CMML is progressing with updates expected later this year Company continues to advance its contemplated merger with Decoy Therapeutics HOUSTON, July 09, 2025 (GLOBE NEWSWIRE) -- Salarius Pharmaceuticals, Inc. (Nasdaq: SLRX) (Salarius or the Company) announces that two animal studies recently published in peer-reviewed journals provide additional insight into the role of inhibiting lysine-demethylase 1 (LSD1 or KDM1A), a well-validated ...

CAMBRIDGE, Mass., July 09, 2025 (GLOBE NEWSWIRE) -- Ernexa Therapeutics (Nasdaq: ERNA), developing innovative cell therapies for the treatment of advanced cancer and autoimmune disease, today announced that it has regained compliance with Nasdaq for continued listing and that its stock will continue to trade on The Nasdaq Stock Market. On July 1, 2025, the company received formal notice from Nasdaq stating that Ernexa has regained compliance with Nasdaq's minimum bid price requirement under Listing Rule 555 ...

HOUSTON, July 09, 2025 (GLOBE NEWSWIRE) -- FibroBiologics, Inc. (Nasdaq: FBLG) ("FibroBiologics"), a clinical-stage biotechnology company with 275+ patents issued and pending with a focus on the development of therapeutics and potential cures for chronic diseases using fibroblasts and fibroblast-derived materials, today announced an advancement in its cartilage repair program. FibroBiologics has confirmed, through both immunohistochemistry and transcriptomic analysis, that the CYWC628 spheroids used in its ...

Tegoprubart to be Used as Immunosuppressive Agent in Cohort C of Sernova’s Phase 1/2 Cell Pouch Bio-hybrid Organ Clinical Trial in Patients with Type 1 DiabetesLONDON, Ontario, July 09, 2025 (GLOBE NEWSWIRE) -- Sernova Biotherapeutics (“Sernova”) (TSX: SVA) (OTCQB: SEOVF) (FSE/XETRA: PSH) today announced a Collaborative Research Agreement with Eledon Pharmaceuticals, Inc. (“Eledon”) (Nasdaq: ELDN) to evaluate Eledon’s immunosuppressive agent tegoprubart (AT-1501) in Sernova’s ongoing Phase 1/2 clinical tria ...

Core Insights - Silexion Therapeutics Corp. announced positive preclinical data for SIL204, demonstrating significant efficacy in human lung cancer cell lines, particularly those with KRAS G12D mutations [1][4][6] - The company is conducting a new study on an untested KRAS mutation, with results expected soon, which could further validate SIL204 as a potential pan-KRAS treatment [2][3] - Silexion's dual-route administration strategy is progressing, with plans to initiate a Phase 2/3 clinical trial in Q2 2026 for KRAS-driven solid tumors [3] Study Findings - The study showed dose-dependent inhibition in lung cancer cells with KRAS G12D mutations, highlighting SIL204's potential as a versatile therapeutic [4][7] - The lipid-conjugated delivery system enhances SIL204's entry into tumor cells, addressing a significant barrier for siRNA technology [5][6] Market Opportunity - KRAS mutations are prevalent in various cancers, with approximately 90% in pancreatic cancers, 45% in colorectal cancers, and 30% in lung cancers, representing a significant market opportunity [8] - The global treatment market for these cancers exceeds US $30 billion annually, with many KRAS variants challenging to target with conventional therapies, underscoring the potential of Silexion's RNAi-based strategy [8][9] Company Overview - Silexion Therapeutics is focused on developing RNA interference therapies for solid tumors driven by KRAS mutations, with SIL204 as its next-generation candidate showing promise in preclinical studies [10]

REDWOOD CITY, Calif., July 09, 2025 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (Nasdaq: JSPR) (Jasper), a clinical stage biotechnology company focused on development of briquilimab, a novel antibody therapy targeting KIT (CD117) to address mast cell driven diseases such as chronic spontaneous urticaria (CSU) and chronic inducible urticaria (CIndU), today announced a corporate reorganization to extend its cash runway, including a workforce reduction of approximately 50%. As part of the reorganization, Edw ...

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements relate to future events or future financial performance, and use words such as "may," "estimate," "could," "expect", "look forward", "believe", "hope" and others. They are based on NORCROSS, Ga., July 09, 2025 (GLOBE NEWSWIRE) -- Galectin Therapeutics Inc. (NASDAQ:GALT), the leading developer of galectin-3-targeted therapeutics for patients with metabolic dysfun ...