FN1

N-acetylcysteine (NAC) shows promise to treat hereditary cystatin C amyloid angiopathy (HCCAA) HCCAA – a subtype of cerebral amyloid angiopathy (CAA) – is an ultra-rare Icelandic amyloid disease due to a mutation in the CST3 gene encoding for the protease inhibitor, cystatin C. HCCAA causes cerebral hemorrhages in asymptomatic carriers of the L68Q variant due to amyloid aggregation with precipitation in brain vessels and tissue. Findings could guide the development of preventative therapies for related CAAs. REYKJAVIK, Iceland, April 2, 2025 /PRNewswire/ -- Icelandic drug discovery and development company Arctic Therapeutics (ATx) today announced the publication of research in JAMA Neurology, which informs a possible therapeutic strategy to treat a rare form of familial dementia. The publication, titled N-Acetylcysteine for hereditary cystatin C amyloid angiopathy (HCCAA), a nonrandomised clinical trial, details a new treatment approach for HCCAA, a rare and fatal genetic brain disorder that primarily affects young Icelandic adults, with the disease process beginning in childhood. The research provides insights into the potential of N-acetylcysteine (NAC) as a treatment for HCCAA, a disease with no currently approved therapeutic options. The study highlights how NAC may reduce disease-associated biomarkers and slow disease progression. The study is the first phase 2a clinical trial to assess the safety and therapeutic potential of high-dose NAC in patients carrying the L68Q-CST3 sequence variation of HCCAA. Results indicate that NAC is well tolerated, reduces disease specific biomarkers and stabilises disease progression, suggesting NAC may offer a preventative strategy against HCCAA. These findings could accelerate the development of preventative therapies for individuals at risk of HCCAA and other forms of CAAs. "These promising results provide hope to HCCAA patients and their families," Dr Ásbjörg Ósk Snorradóttir, lead author of the study explained. Senior author, Dr. Hákon Hákonarson, director of the Center for Applied Genomics at the Children's Hospital of Philadelphia and founder of Arctic Therapeutics added: "This study provides the first clinical evidence that NAC, by breaking disulfide bonds and other aberrant molecular interactions, can disrupt cystatin C oligomers and prevent amyloid aggregation that ultimately accumulates in brain vessels. By targeting the root biochemical process behind HCCAA pathology, NAC may ultimately offer a novel disease-modifying approach, not only for this devastating condition, but potentially for other forms of CAAs and protein aggregation disorders where toxic oligomers are central to disease progression." The open-label phase 2a clinical trial was conducted at Iceland's Landspitali University Hospital from March 2019 to December 2021. A total of 17 participants were enrolled, all confirmed carriers of the L68Q-CST3 mutation. The study assessed: Safety & tolerability: NAC was well tolerated, with no drug-related severe adverse events. Five participants experienced cerebral hemorrhages, but none resulted in permanent neurological damage. Biomarker reduction: Significant reductions were observed in collagen IV, fibronectin, vimentin, and SMAD levels—key indicators of disease progression. Plasma & skin biomarker changes: NAC was shown to increase reduced glutathione levels, a protective antioxidant, and reduce high-molecular-weight cystatin C aggregates, which are linked to amyloid buildup in the brain. Cognitive stability: No measurable decline in cognitive function was observed during the study period. Building on these findings, as well as internal in-vitro data that demonstrates that NAC-amide (NACA) has more than 10-fold higher potency in preventing or dispersing amyloid complex aggregates than NAC and increased ability to penetrate the blood brain barrier and get into cells involved in the disease pathogenesis, ATx has launched a European Medicines Agency-approved registration study to monitor safety, tolerability, and efficacy using NACA in patients with HCCAA. For more details, read the full study in JAMA Neurology: Original Investigation. About Arctic Therapeutics Arctic Therapeutics is an Icelandic drug discovery and development company established in 2015, which has licensed intellectual property from Children's Hospital of Philadelphia. ATx leverages the transformative power of applied genomics, steering the course of drug development towards safer, more effective treatments for some of the world's most challenging diseases. The company has operations in Iceland, the US and multiple collaborations across Europe. For more information, please visit: and follow us on LinkedIn For media inquires Gulli Arnason, Chief Strategy Officer [email protected] +354 660 0053 This information was brought to you by Cision The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Following positive results from the Phase 1 dose escalation study, BND-22 (SAR444881) is now being tested in Phase 2 trials as monotherapy for patients with cholangiocarcinoma and in combination with cetuximab in patients with non-small cell lung cancer (NSCLC) and colorectal cancer (CRC). MISGAV, Israel, March 3, 2025 /PRNewswire/ -- Biond Biologics Ltd. ("Biond" or the "Company"), a private clinical-stage biopharmaceutical company developing novel therapies for cancer, today announced that it would regain full rights to BND-22 (SAR444881) from Sanofi. Sanofi is returning the rights of BND-22 as part of their broader R&D prioritization to focus on programs that support the company's strategy. Biond and Sanofi, are working together to complete full transfer of the BND-22 program to Biond. In January 2021, Biond entered into an exclusive worldwide license agreement with Sanofi for the development and commercialization of BND-22. As part of the agreement, Biond collaborated with Sanofi to initiate a first-in-human Phase 1 study (BND-22-001, NCT04717375), designed to evaluate the safety and tolerability of BND-22, both as a monotherapy and in combination with the approved cancer therapies cetuximab and pembrolizumab. The Phase 1 dose-escalation study was successfully completed and demonstrated a favorable safety profile across all patient groups. More specifically, data showed that BND-22 was well-tolerated and exhibited anti-tumor activity in heavily pretreated patients. Consistent with preclinical findings, a dose-dependent upregulation of activation markers was observed in monocytes and ILT2-expressing T and natural killer (NK) cell subsets. Notably, several confirmed clinical responses to both monotherapy and combination therapies regimens, were reported during the dose-escalation phase. These findings highlight the potential of BND-22 to address critical unmet needs in oncology. Building on these encouraging results, Sanofi initiated enrollment for a Phase 2 dose-optimization and expansion study (BND-22-001, NCT04717375). The study was designed to evaluate BND-22 as monotherapy for patients with cholangiocarcinoma and in combination with cetuximab for patients with non-small cell lung cancer (NSCLC) or colorectal cancer (CRC). For more information about the trial, please visit (Trial Identifier: NCT04717375). "As part of the agreement between Biond and Sanofi, Biond will regain full rights to BND-22, including access to the clinical data generated by Sanofi," said Dr. Tehila Ben Moshe, Biond's CEO and co-founder. "Sanofi has been an outstanding partner, and together, we have made remarkable progress in advancing the clinical development of BND-22. We remain committed to the continued development of BND-22 program, whether independently or in partnership with strategic collaborators". "We are encouraged by the data demonstrated so far for BND-22 in the dose-escalation and dose-expansion study", said Dr. Natalia Ashtamker, Biond's VP of Clinical Development. "We intend to continue treating patients who are benefiting from BND-22 treatment in the BND-22-001 study and are looking forward to the initiation of a Phase 2 biomarker study of BND-22 in combination with anti-PD-1 therapy". About BND-22 BND-22 is a humanized IgG4 antagonist antibody targeting the ILT2 receptor, developed for the treatment of solid tumors. ILT2 is an inhibitory immuno-modulating receptor expressed on both innate and adaptive immune cells. It binds to major histocompatibility complex (MHC) class I molecules, including HLA-G, an immunosuppressive protein expressed by various tumor types. Preclinical studies have demonstrated that BND-22 exerts broad anti-tumor effects by disrupting ILT2-mediated "do not eat me" signals in macrophages and activating NK and CD8+ lymphocytes. BND-22-001, a Phase 1/2 multicenter, open-label, dose-escalation, dose-expansion and dose optimization study enrolled patients with advanced solid tumors known to express HLA-G. The study included evaluations of BND-22 as monotherapy in cholangiocarcinoma and in combination with cetuximab in patients with non-small cell lung cancer (NSCLC) and colorectal cancer (CRC). Additionally, a future Phase 2 biomarker trial will explore BND-22 in combination with an anti-PD-1 agent in NSCLC, CRC and ovarian cancers. (Trial Identifier: NCT04717375). About Biond Biologics Biond Biologics is a drug discovery and development company focused on developing innovative therapies for novel oncology targets identified through research of immunoregulatory pathways. In parallel, it is developing a dedicated platform to facilitate the intracellular delivery of payloads, including biologics. Biond's clinical pipeline includes BND-22, a multi-cell checkpoint inhibitor targeting ILT2, and BND-35, a unique anti-ILT3/LILRB4 blocking antibody that drives the suppressive tumor microenvironment to become more pro-inflammatory. BND-35 also augments the Antibody dependent cellular cytotoxicity (ADCC) and Antibody dependent cellular phagocytosis (ADCP) activity of cancer-targeted therapies. BND-35 blocks the interaction between ILT3 and various ligands, including fibronectin, which can serve as a biomarker for patient selection. BND-35 entered a Phase 1 trial in May 2024. Biond is also developing BND-67, a first-in-class antibody, that specifically prevents the shedding of CD28 from T cells. Blocking CD28 shedding, drives prolonged T cell activation and enables safe and sustained immune responses. This unique approach can overcome resistance in patients treated with T cell-targeting therapies, such as anti-PD1/PDL1 blockers. BND-67 is expected to enter a phase 1 trial in 2026. In addition to its pipeline of immunotherapy agents, Biond is also developing INspire, a revolutionary platform that allows pH-specific delivery of various molecules, including biological drugs and small molecules into cancer cells. Biond was founded in 2016, by Tehila Ben Moshe, Ph.D., Ori Shilo, and a team of accomplished scientists and drug developers from the Israel biopharmaceutical industry. . Company contact: Tal Sines VP Business Development Biond Biologics Ltd. +972-4 884 4337 [email protected] SOURCE Biond Biologics Ltd. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

BOSTON, Feb. 26, 2025 (GLOBE NEWSWIRE) -- Pyxis Oncology, Inc. (Nasdaq: PYXS), a clinical-stage company developing next-generation therapeutics for difficult-to-treat cancers, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to PYX-201 for the treatment of adult patients with recurrent or metastatic head and neck squamous cell carcinoma (R/M HNSCC) whose disease has progressed following treatment with platinum-based chemotherapy and an anti-PD-(L)1 antibody. PYX-201 is a first-in-concept antibody-drug conjugate (ADC) that uniquely targets Extradomain-B Fibronectin (EDB+FN), a non-cellular structural component within the tumor extracellular matrix (ECM), which is highly expressed in various tumor types. “Receiving Fast Track designation for PYX-201 from the FDA marks a significant milestone for Pyxis Oncology, recognizing our potential to address the significant medical need in R/M HNSCC. This designation underscores the urgency of bringing differentiated treatment options to patients and will help accelerate the development of PYX-201 as we actively recruit patients for our trial,” said Lara S. Sullivan, M.D., President and Chief Executive Officer. “We look forward to working with the FDA to advance this promising therapy as efficiently as possible.” Fast Track designation is an FDA program intended to facilitate and expedite the development and review of new drugs in the U.S. for the treatment of a serious or life-threatening condition. To qualify for this designation, there must be clear data demonstrating the drug has potential to address unmet medical need in the designated condition. About Head and Neck Squamous Cell Carcinoma (HNSCC) Head and Neck Cancer (HNC) is the sixth most common cancer in the world, with 1,464,550 new cases and 487,993 deaths from HNC globally 1 . Squamous Cell Carcinoma presents as the most common subtype and is derived from the mucosal lining of the oral cavity, pharynx and larynx. Almost 50% of cases progress to recurrent or metastatic cancer post-initial treatment, presenting patients with a median overall survival of less than a year. The overall incidence of HNSCC is expected to rise, with a predicted 30% increase annually by 2030 2 . The increase has been associated with multiple factors, including but not limited to tobacco use, alcohol consumption, a rise in HPV infections, and other environmental catalysts. With limited development outside of immunotherapy in the last decade, HNSCC remains one of the most difficult to treat carcinomas, highlighting the unmet need. 1 Zhou T, Huang W, Wang X, Zhang J, Zhou E, Tu Y, et al. Global burden of head and neck cancers from 1990 to 2019. iScience . 2024;27:109282 United States ; 2 Gormley, M., Creaney, G., Schache , A. et al. Reviewing the epidemiology of head and neck cancer: definitions, trends and risk factors. Br Dent J 233, 780–786 (2022). About PYX-201 PYX-201, an antibody-drug conjugate (ADC) with a microtubule inhibitor (optimized auristatin) payload that uniquely targets Extradomain-B Fibronectin (EDB+FN), a non-cellular structural component of the tumor extracellular matrix (ECM), is the company’s lead clinical drug candidate. Two PYX-201 trials are now actively recruiting. One trial, PYX-201-101, is designed to evaluate PYX-201 as monotherapy in patients with R/M HNSCC. A second trial, PYX-201-102, is evaluating PYX-201 in combination with Merck’s anti-PD-1 therapy, KEYTRUDA ® (pembrolizumab), in patients with R/M HNSCC and other advanced solid tumors. The combination trial is part of a recently announced Clinical Trial Collaboration Agreement with Merck (known as MSD outside of the US and Canada). KEYTRUDA ® is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA. About Pyxis Oncology, Inc. Pyxis Oncology, Inc. is a clinical stage company focused on defeating difficult-to-treat cancers. The company is efficiently building next generation therapeutics that hold the potential for monotherapy and combination indications. PYX-201, an antibody-drug conjugate (ADC) that uniquely targets EDB+FN, a non-cellular structural component of the tumor extracellular matrix, is being evaluated in ongoing Phase 1 clinical studies in multiple types of solid tumors. PYX-201 is designed to directly kill cancer cells and to address factors in the microenvironment that enable the uncontrolled proliferation and immune evasion of malignant tumors. To learn more, visit www.pyxisoncology.com or follow us on X (formerly known as Twitter) and LinkedIn. Forward Looking Statements This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. These statements are often identified by the use of words such as “anticipate,” “believe,” “can,” “continue,” “could,” “estimate,” “expect,” “intend,” “likely,” “may,” “might,” “objective,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “to be,” “will,” “would,” or the negative or plural of these words, or similar expressions or variations, although not all forward-looking statements contain these words. We cannot assure you that the events and circumstances reflected in the forward-looking statements will be achieved or occur and actual results could differ materially from those expressed or implied by these forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those identified herein, and those discussed in the section titled “Risk Factors” set forth in Part II, Item 1A. of the Company’s Quarterly Report on Form 10-Q filed with SEC on November 12, 2024, and our other filings, each of which is on file with the Securities and Exchange Commission. These risks are not exhaustive. New risk factors emerge from time to time, and it is not possible for our management to predict all risk factors, nor can we assess the impact of all factors on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. In addition, statements that “we believe” and similar statements reflect our beliefs and opinions on the relevant subject. These statements are based upon information available to us as of the date hereof and while we believe such information forms a reasonable basis for such statements, such information may be limited or incomplete, and our statements should not be read to indicate that we have conducted an exhaustive inquiry into, or review of, all potentially available relevant information. These statements are inherently uncertain, and investors are cautioned not to unduly rely upon these statements. Except as required by law, we undertake no obligation to update any forward-looking statements to reflect events or circumstances after the date of such statements. Source: Pyxis Oncology, Inc. Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.