Latest news with #orphanDrug
Yahoo
08-07-2025
- Business
- Yahoo
U.S. FDA Grants Orphan Drug Designation to Adcentrx Therapeutics' ADRX-0405 STEAP1 ADC for Gastric Cancer
Orphan drug designation highlights the potential for ADRX-0405 to address the high unmet need in gastric cancer SAN DIEGO, July 8, 2025 /PRNewswire/ -- Adcentrx Therapeutics ("Adcentrx"), a clinical-stage biotechnology company redefining Antibody-Drug Conjugate (ADC) therapies for cancer treatment and other life-threatening diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ADRX-0405, for the treatment of patients with gastric cancer. ADRX-0405 is a STEAP1 ADC being evaluated in the Phase 1a portion of an ongoing Phase 1a/b clinical trial (NCT06710379) for the treatment of select advanced solid tumors, including metastatic castration resistant prostate cancer, gastric cancer, and non-small cell lung cancer. While STEAP1 is primarily associated with prostate cancer, there is a meaningful amount of target expression in gastric cancer, making this a potential indication of interest for future clinical development. "Receiving orphan drug designation from FDA is a notable milestone for Adcentrx and reinforces the potential for ADRX-0405 to improve the lives of patients with gastric cancer," said Hui Li, Ph.D., Founder and Chief Executive Officer of Adcentrx. "We are encouraged by the progress of our Phase 1a trial and look forward to further evaluating the safety, tolerability and anti-tumor activity of ADRX-0405 in gastric and other cancers." Gastric cancer, or stomach cancer, is a serious malignancy that develops in the stomach lining and is often diagnosed at advanced stages. The American Cancer Society estimates there will be 30,300 new cases of gastric cancer in the U.S. in 2025, meeting FDA's criteria for a rare disease. The orphan drug designation is a program designed to stimulate the development of treatments for rare diseases, defined as conditions affecting fewer than 200,000 people in the U.S. Benefits of this designation include access to grant funding and scientific assistance, tax credits for qualified clinical trials, waiver of Prescription Drug User Fee Act (PDUFA) application fees, and the potential for seven years of market exclusivity following regulatory approval. About ADRX-0405ADRX-0405 is a clinical-stage next-generation ADC targeting six-transmembrane epithelial antigen of the prostate 1 (STEAP1), a cell surface protein that is upregulated in prostate cancer and certain other cancers with limited expression in normal healthy tissue. The ADC is composed of a humanized IgG1 antibody coupled with a novel topoisomerase inhibitor linker-payload through Adcentrx's innovative i-Conjugation® technology platform – a core component in the design of the company's ADCs. The platform utilizes a cleavable linker and stable conjugation chemistry to enhance payload delivery. This novel technology enables a highly stable ADC with a drug-antibody ratio of eight (DAR 8) to maximize payload delivery to solid tumors. ADRX-0405 preclinical studies have demonstrated its favorable pharmacokinetics, safety profile, and significant efficacy across multiple animal tumor models. ADRX-0405 is currently being evaluated in a Phase 1a/b clinical trial. For more information about the ADRX-0405 Phase 1a/b clinical trial, please refer to the Study ID NCT06710379 on About Adcentrx TherapeuticsAdcentrx is a biotechnology company focused on accelerating breakthroughs in protein conjugate therapeutic development for cancer and other life-threatening diseases. Adcentrx has pioneered the development of an ADC technology platform addressing key components of protein conjugate design to solve challenges typically seen in ADCs. Adcentrx is developing a robust pipeline including two clinical-stage ADCs and multiple preclinical ADCs, all with first-in-class and best-in-class potential. For more information about Adcentrx and its innovative ADC technologies, please visit Contact Information:Investor Relationsir@ View original content to download multimedia: SOURCE Adcentrx Therapeutics
Yahoo
06-07-2025
- Business
- Yahoo
FDA Grants Orphan Drug Designation To Sanofi's (SNY) Riliprubart
Sanofi (NASDAQ:SNY) is one of the . On June 25, Sanofi (NASDAQ:SNY) announced that the FDA granted orphan drug designation to Riliprubart for treating antibody-mediated rejection in solid organ transplantation. The FDA grants orphan drug designation to drugs aimed at treating rare diseases or conditions affecting fewer than 200,000 people in the US. This is a significant milestone for Sanofi (NASDAQ:SNY) as it provides benefits including tax credits, user fee waivers, and market exclusivity upon approval. Dozens of pharmaceutical capsules piled on top of one another to show the scale of the company's drug contributions to the industry. Riliprubart is currently being evaluated in multiple clinical trials across different indications, including transplant and neurology. The phase 2 study is underway to assess its efficacy in kidney transplant recipients. Sanofi (NASDAQ:SNY) is also conducting two phase 3 trials investigating Riliprubart in chronic inflammatory demyelinating polyneuropathy. Sanofi (NASDAQ:SNY) is a leading healthcare company headquartered in France. It focuses on improving patient health through the research, development, manufacturing, and marketing of a wide range of therapeutic solutions. While we acknowledge the potential of SNY as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: The Best and Worst Dow Stocks for the Next 12 Months and 10 Unstoppable Stocks That Could Double Your Money. Disclosure: None. Error while retrieving data Sign in to access your portfolio Error while retrieving data Error while retrieving data Error while retrieving data Error while retrieving data
Yahoo
06-07-2025
- Business
- Yahoo
Sanofi's (NASDAQ:SNY) Riliprubart Granted Orphan Frug Designation in Japan
Sanofi (NASDAQ:SNY) is one of the 11 Best 52-Week Low Stocks to Buy Right Now. On June 30, Sanofi (NASDAQ:SNY) announced that its Riliprubart has been granted orphan drug designation in Japan for chronic inflammatory demyelinating polyneuropathy. Riliprubart is a monoclonal antibody that specifically inhibits activated C1s in the classical complement pathway. The treatment is designed to block a very specific part of the immune system's response. Orphan drug designation is a special status granted by regulatory bodies for medicines that target rare diseases or conditions where there is a high unmet medical need. Dozens of pharmaceutical capsules piled on top of one another to show the scale of the company's drug contributions to the industry. This is a significant achievement for Sanofi (NASDAQ:SNY) because, despite existing treatments, around 30% of patients do not respond adequately, leaving a significant unmet medical need. Sanofi (NASDAQ:SNY) presented 76-week long-term data from a phase 2 clinical trial at the Peripheral Nerve Society meeting in Edinburgh. The results suggest sustained efficacy and safety of Riliprubart in a broad range of CIDP patients. Management noted that two phase 3 studies are underway to further evaluate the treatment. While we acknowledge the potential of SNY as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: The Best and Worst Dow Stocks for the Next 12 Months and 10 Unstoppable Stocks That Could Double Your Money. Disclosure: None. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
30-06-2025
- Business
- Yahoo
Sanofi's Experimental Transplant Rejection Drug Gets FDA Orphan Tag
Sanofi SNY announced that the FDA has granted orphan drug designation to its investigational drug, riliprubart, for the treatment of antibody-mediated rejection (AMR) in solid organ transplantation. Orphan drug designation is granted by the FDA to therapies intended for the treatment of rare diseases or conditions affecting fewer than 200,000 people in the United States. The goal is to encourage drug development for rare diseases or conditions by offering incentives such as tax credits for clinical testing, waiver of certain fees and most importantly, seven years of market exclusivity upon approval. For Sanofi, this designation provides riliprubart with important regulatory and financial advantages as it advances through clinical development for AMR in solid organ transplantation — a serious complication where the immune system attacks the donated organ. Currently, there are no FDA-approved therapies for this condition. The designation may help accelerate progress and reduce the development burden as Sanofi works to address this critical unmet need in transplant medicine. Shares of Sanofi have outperformed the industry year to date, as seen in the chart below. Image Source: Zacks Investment Research An investigational IgG4 humanized monoclonal antibody, riliprubart is designed to selectively inhibit activated C1s in the classical complement pathway of the innate immune system. The drug is currently being evaluated in a mid-stage study for AMR in kidney transplant recipients across two patient groups — those at risk of developing rejection and those already experiencing active AMR. Apart from AMR, Sanofi is also evaluating riliprubart in two late-stage studies — MOBILIZE and VITALIZE — for a rare neurological disorder called chronic inflammatory demyelinating polyneuropathy (CIDP). While the MOBILIZE study targets patients for whom standard-of-care treatments do not work, the VITALIZE study compares the drug against intravenous immunoglobulin (IVIg). CIDP is a rare disorder affecting the peripheral nervous system, marked by progressive muscle weakness and sensory loss. Notably, riliprubart has also received orphan drug designation in both the United States and EU for its prospective use in CIDP, highlighting its potential across multiple immune-mediated diseases. Sanofi currently carries a Zacks Rank #3 (Hold). Sanofi price | Sanofi Quote Some better-ranked stocks from the sector are Amarin Corporation AMRN, Immunocore IMCR and Agenus AGEN. While AMRN and IMCR sport a Zacks Rank #1 (Strong Buy) each at present, AGEN carries a Zacks Rank #2 (Buy). You can see the complete list of today's Zacks #1 Rank stocks here. In the past 60 days, loss per share estimates for Amarin's 2025 have improved from $5.01 to $2.50. Loss per share estimates for 2026 have narrowed from $3.84 to $1.78 during the same period. AMRN stock has surged nearly 65% year to date. Amarin's earnings beat estimates in two of the trailing four quarters, met the mark once and missed in the other, delivering an average surprise of 29.11%. In the past 60 days, loss per share estimates for Immunocore's 2025 have improved from $1.50 to 86 cents. Loss per share estimates for 2026 have narrowed from $1.68 to $1.33 during the same period. IMCR stock has gained nearly 9% year to date. Immunocore's earnings beat estimates in three of the trailing four quarters and missed the mark once, delivering an average surprise of 76.18%. In the past 60 days, Agenus' bottom-line estimates for 2025 have significantly improved from a loss of $3.46 per share to earnings of $1.56. During the same timeframe, estimates for 2026 loss per share have narrowed from $3.91 to $1.99. AGEN stock has soared 83% so far this year. Agenus' earnings beat estimates in two of the trailing four quarters and missed the mark on the other two occasions, delivering an average negative surprise of 22.71%. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Sanofi (SNY) : Free Stock Analysis Report Agenus Inc. (AGEN) : Free Stock Analysis Report Amarin Corporation PLC (AMRN) : Free Stock Analysis Report Immunocore Holdings PLC Sponsored ADR (IMCR) : Free Stock Analysis Report This article originally published on Zacks Investment Research ( Zacks Investment Research Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
30-06-2025
- Health
- Yahoo
Press Release: Riliprubart granted orphan drug designation in Japan for chronic inflammatory demyelinating polyneuropathy
Riliprubart granted orphan drug designation in Japan for chronic inflammatory demyelinating polyneuropathy Two phase 3 studies are currently underway testing riliprubart in people with CIDP as a potential first-in-class treatment Japanese Ministry of Health, Labour and Welfare decision adds to similar designations in the US and Europe, underscoring global regulatory recognition of the potential for riliprupart to address significant unmet medical needs for people living with this rare neurological condition Paris, June 30, 2025. The Ministry of Health, Labour and Welfare (MHLW) in Japan has granted orphan drug designation to riliprubart, a monoclonal antibody that selectively inhibits activated C1s in the classical complement pathway for people with chronic inflammatory demyelinating polyneuropathy (CIDP). Despite available therapies, many CIDP patients are left with residual symptoms, including weakness, numbness, and fatigue that can lead to long-term morbidity and diminished quality of life. Approximately 30% of people with CIDP do not respond to standard therapies. The MHLW grants orphan drug designation to medicines that address rare medical diseases or conditions with unmet medical needs. There are currently approximately 4,000 people diagnosed with CIDP in Japan. Global Head of Neurology Development, Sanofi'The orphan drug designation of riliprubart for people living with CIDP in Japan underscores our commitment to applying our deep understanding of the immune system to address rare neurological disorders with significant unmet medical needs. While CIDP therapies exist, many individuals continue to experience debilitating symptoms, including pain, fatigue and weakness. Our ongoing development of riliprubart reflects our dedication to challenging the status quo in neurology with the goal of improving people's lives.' Long-term, 76-week sustained efficacy and safety data from riliprubart's phase 2 study were presented at the Peripheral Nerve Society meeting in Edinburgh, UK on May 17-20, 2025. Findings suggest a potential sustained long-term benefit provided by riliprubart across a broad spectrum of participants with CIDP. Riliprubart is currently being tested in two separate phase 3 studies: MOBILIZE in patients refractory to standard of care (clinical study identifier: NCT06290128) and VITALIZE in IVIg-treated patients (clinical study identifier: NCT06290141). About riliprubartSAR445088 (riliprubart) is an IgG4 humanized monoclonal antibody that selectively inhibits activated C1s in the classical complement pathway of the innate immune system. By blocking C1s, riliprubart has the potential to inhibit key inflammatory mechanisms that drive demyelination and axonal damage in CIDP. Riliprubart is currently under clinical investigation, and its safety and efficacy have not been evaluated by any regulatory authority. For more information on riliprubart clinical studies, please visit CIDPCIDP is a rare neurological condition that causes progressive weakness and sensory impairment in the arms and legs. CIDP occurs when the body's immune system attacks the myelin sheaths around nerve cells in the peripheral nervous system. Timely diagnosis of CIDP is important because it allows for appropriate treatment, which is essential to preventing long-term disability. However, despite available therapies, many individuals are left with residual symptoms, including weakness, numbness, and fatigue that can lead to long-term morbidity and diminished quality-of-life. Approximately 30% of people with CIDP do not respond to standard therapies. In people with CIDP who do respond, about 70% of the response is considered incomplete. Less than one-third of people with CIDP remain in remission without continued therapy. About Sanofi Sanofi is an R&D driven, AI-powered biopharma company committed to improving people's lives and delivering compelling growth. We apply our deep understanding of the immune system to invent medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more. Our team is guided by one purpose: we chase the miracles of science to improve people's lives; this inspires us to drive progress and deliver positive impact for our people and the communities we serve, by addressing the most urgent healthcare, environmental, and societal challenges of our time. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY Media RelationsSandrine Guendoul | +33 6 25 09 14 25 | Berland | +1 215 432 0234 | Léo Le Bourhis | +33 6 75 06 43 81 | Victor Rouault | +33 6 70 93 71 40 | Timothy Gilbert | +1 516 521 2929 | Ubaldi | +33 6 30 19 66 46 | Investor RelationsThomas Kudsk Larsen |+44 7545 513 693 | Alizé Kaisserian | +33 6 47 04 12 11 | Lauscher | +1 908 612 7239 | Browne | +1 781 249 1766 | Nathalie Pham | +33 7 85 93 30 17 | Elgoutni | +1 617 710 3587 | Thibaud Châtelet | +33 6 80 80 89 90 | Li | +33 6 84 00 90 72 | Sanofi forward-looking statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions, and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words 'expects', 'anticipates', 'believes', 'intends', 'estimates', 'plans' and similar expressions. Although Sanofi's management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the fact that product candidates if approved may not be commercially successful, the future approval and commercial success of therapeutic alternatives, Sanofi's ability to benefit from external growth opportunities, to complete related transactions and/or obtain regulatory clearances, risks associated with intellectual property and any related pending or future litigation and the ultimate outcome of such litigation, trends in exchange rates and prevailing interest rates, volatile economic and market conditions, cost containment initiatives and subsequent changes thereto, and the impact that global crises may have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under 'Risk Factors' and 'Cautionary Statement Regarding Forward-Looking Statements' in Sanofi's annual report on Form 20-F for the year ended December 31, 2024. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements. All trademarks mentioned in this press release are the property of the Sanofi group. Attachment Press ReleaseError in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
