Key Takeaways KROS is advancing KER-065 for DMD, with FDA orphan drug status and a phase II trial planned for early 2026.DYN reported positive data for z-rostudirsen, showing dystrophin gains and functional improvements in DMD.DYN held $791.9M in cash as of Sept. 2025, funding operations into Q3 2027, alongside multiple programs.Keros Therapeutics (KROS) is a clinical-stage biopharmaceutical company developing innovative therapies for patients with disorders caused by abnormal signaling within the transform ...

Summary of Cardiff Oncology Update Call Company Overview - **Company**: Cardiff Oncology (NasdaqCM:CRDF) - **Focus**: Development of onvansertib, a highly specific oral PLK1 inhibitor, primarily for RAS-mutated metastatic colorectal cancer (mCRC) patients Key Points Management Transition - Cardiff Oncology announced a management transition to better position the company for late-stage development of onvansertib [5][6] - Mani Mohindru appointed as Interim CEO, emphasizing the need for leadership aligned with the company's evolving operational and financial needs [4][5] Clinical Trial Updates - Promising phase 2 data from the CRDF-004 trial for onvansertib in first-line RAS-mutated mCRC patients was discussed [5][7] - The trial evaluated onvansertib in combination with standard care regimens (FOLFIRI plus bevacizumab or FOLFOX plus bevacizumab) [8] - Dose-dependent benefits observed, particularly with the 30 mg dose of onvansertib combined with FOLFIRI plus bevacizumab, showing an overall objective response rate of 72.2% compared to 43.2% with FOLFOX and 42.1% with FOLFIRI alone [10][11] Efficacy and Safety Data - Median progression-free survival (PFS) has not yet been reached in the onvansertib arms, indicating extended benefit compared to a median PFS of about 11 months for standard care [10] - The PFS hazard ratio was reported at 0.37 for the 30 mg onvansertib plus FOLFIRI bev arm, indicating a significant reduction in disease progression risk [11] - Safety profile remains favorable, with no unexpected toxicities reported; grade 3 or higher adverse events were infrequent [12] Future Plans - Cardiff plans to initiate a registrational study later in 2026, comparing onvansertib plus FOLFIRI to standard care regimens [12][14] - The study protocol will likely include both FOLFIRI and FOLFOX in the control arm to provide comprehensive data for regulatory review [13] - Anticipation of more mature clinical data from the CRDF-004 study by mid-2026 [14] Strategic Considerations - Discussions regarding potential partnerships to support the phase 3 trial and broaden development beyond initial indications are ongoing [33] - The company is focused on building a leadership team with experience in late-stage development and commercialization [76] Additional Insights - The transition in leadership is not due to issues with onvansertib but rather a strategic move to capitalize on promising data [6] - The focus on durability of response is emphasized, with stable disease being a significant factor in treatment efficacy [24][25] - The company is committed to maintaining operational continuity during the management transition [7] Conclusion Cardiff Oncology is positioned for significant growth with onvansertib, supported by promising clinical data and a strategic management transition aimed at enhancing its late-stage development efforts. The upcoming registrational study and potential partnerships are critical steps in advancing the drug's market potential.

Core Insights - INmune Bio, Inc. has made significant progress in 2025, focusing on developing therapies for neuroinflammatory and immunologic diseases, particularly through its programs XPro™ for Alzheimer's disease and CORDStrom™ for Recessive Dystrophic Epidermolysis Bullosa (RDEB) [1][10] CORDStrom™ Program - CORDStrom™, an allogeneic umbilical cord-derived mesenchymal stromal cell therapy for RDEB, achieved substantial progress in 2025, including successful completion of three commercial pilot-scale runs in preparation for regulatory filings [2] - The company plans to submit a Marketing Authorization Application (MAA) to the UK's Medicines and Healthcare products Regulatory Agency (MHRA) by mid-summer 2026, followed by a Biologics License Application (BLA) to the U.S. FDA towards the end of 2026 [2][3] XPro™ Program - XPro™ demonstrated potential as a differentiated therapy for Alzheimer's disease, with a Phase 2 study (MINDFuL) revealing meaningful signals in a predefined subpopulation of amyloid-positive early AD patients [4][6] - In this subpopulation, XPro™ showed an effect size of 0.27 on the EMACC cognitive scale after 6 months, which compares favorably to the effect sizes of approved anti-amyloid therapies [6][7] - The results support XPro's proposed mechanism of action, which involves selectively inhibiting soluble TNF to reduce neuroinflammation while preserving beneficial TNF signaling [8] Financial Position - As of September 30, 2025, INmune Bio had approximately $27.7 million in cash and cash equivalents, positioning the company well for continued execution and enabling it to achieve key milestones through year-end 2026 [9]

Core Insights - Propanc Biopharma, Inc. has filed its fourth new provisional patent application in two months, focusing on innovative formulations of pancreatic proenzymes, which are critical for addressing challenges in stability and transport for biomedical applications [1][2] - The company aims to double its intellectual property portfolio from approximately 90 to over 200 patents, covering various aspects of its proenzyme technology, which targets and prevents metastasis from solid tumors [2][3] - Propanc's lead asset, PRP, is designed to selectively attack cancer stem cells and circulating tumor cells while sparing healthy tissue, offering a potential long-term therapy with reduced toxicity compared to conventional treatments [3][4] Company Developments - The recent patent applications are part of a strategy to secure leadership in a field with significant unmet medical needs, particularly for aggressive cancers with limited treatment options [2][3] - Preparations for a Phase 1b First-In-Human study of PRP in advanced cancer patients are advancing rapidly, with expectations for further updates on the clinical pathway and broader pipeline soon [3] Industry Context - Propanc is developing a novel approach to cancer treatment by targeting cancer stem cells through proenzyme activation, which addresses the underlying drivers of cancer proliferation and spread [4]

MORRISVILLE, N.C., Jan. 27, 2026 (GLOBE NEWSWIRE) -- Liquidia Corporation (NASDAQ: LQDA), a biopharmaceutical company revolutionizing care for patients with challenging respiratory and vascular diseases, announced today the company will present three posters at the Pulmonary Vascular Research Institute (PVRI) 2026 Annual Congress to be held January 28 through February 1, 2026, in Dublin, Ireland. The presentations highlight clinical data in pulmonary arterial hypertension (PAH) and pulmonary hypertension as ...

Arcutis Biotherapeutics (ARQT) announced that on January 23, 2026, the Company and Kowa Pharmaceuticals America mutually agreed to terminate their promotion agreement. The agreement covered sales and promotion of ZORYVE by Kowa to primary care physicians and pediatricians in the United States. Following the termination, Arcutis plans to assume responsibility for sales and promotion of ZORYVE in the pediatric and primary care settings. The Company is finalizing its plans for promotion to these clinicians an ...

LANGHORNE, Pa.--(BUSINESS WIRE)--Savara Inc. (Nasdaq: SVRA) (the Company), a clinical stage biopharmaceutical company focused on rare respiratory diseases, today announced the amendment of its loan and security agreement with Hercules Capital, Inc. (NYSE:HTGC), strengthening its balance sheet and liquidity. Under the terms of the amended loan agreement, up to an additional $75 million will become available upon FDA's approval of MOLBREEVI, the Company's investigational therapy in autoimmune pul. ...

AMENDMENT NO. 2 TO FORM S-1 REGISTRATION STATEMENT Under The Securities Act of 1933 ___________________________ Polaryx Therapeutics, Inc. (Exact name of registrant as specified in its charter) ___________________________ (State or other jurisdiction of incorporation or organization) As filed with the Securities and Exchange Commission on January 27, 2026 Registration No. 333-291681 UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 (Primary Standard Industrial Classification Code Numbe ...

“On Track with HCM” Features Practical Insights for Managing Everyday Life with the Disease, Inspired by the Experience with HCM of McLaughlin-Levrone’s Father, Willie McLaughlin SOUTH SAN FRANCISCO, Calif., Jan. 27, 2026 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced the launch of “On Track with HCM,” demonstrating the company’s long-standing commitment to the HCM community. The campaign features four-time Olympic gold medalist Sydney McLaughlin-Levrone, and her father, three ...

BRIDGEWATER, N.J., Jan. 27, 2026 (GLOBE NEWSWIRE) -- Amneal Pharmaceuticals, Inc. (NASDAQ: AMRX) (“Amneal” or the “Company”), today announced that the Company will release its fourth quarter and full year 2025 financial results on Friday, February 27, 2026, prior to market open. The Company will host an audio webcast at 8:30 a.m. ET. The financial results and live webcast will be accessible through the Investor Relations section of the Company's website at https://investors.amneal.com. Individuals may regis ...