Latest news with #OrphanDrugDesignation
Yahoo
4 days ago
- Business
- Yahoo
RemeGen's Telitacicept (RC18) Received Orphan Drug Designation from EMA for Myasthenia Gravis
YANTAI, China, June 17, 2025 /PRNewswire/ -- On June 16, 2025, RemeGen Co., Ltd. ("RemeGen", stock symbols: announced that telitacicept (RC18; brand name: 泰爱®) has received Orphan Drug Designation (ODD) from European Medicines Agency (EMA) for the treatment of Myasthenia Gravis (MG), marking a key milestone achieved in its global development. Telitacicept now is the first dual-target biologic drug for MG with ODD from both FDA and EMA worldwide. The designation, granted based on telitacicept's significant benefits in treating the life-threatening rare disease of MG, will provide advantages to this novel agent on protocol assistance, regulatory fee reductions/waivers and up to 10 years of market exclusivity, thereby accelerating the clinical development, registration and drug approval process in Europe and its availability among MG patients. MG is an acquired antibody-mediated rare autoimmune disorder affecting the neuromuscular junction. According to the Myasthenia Gravis Foundation of America (MGFA) and various studies, its global prevalence is estimated to be 15-25 per 100,000, meeting EMA's definition of the rare disease (the prevalence of less than 5 in 10,000 in the EU). Though the current treatment options (including cholinesterase inhibitors, glucocorticoids, immunosuppressants, intravenous immunoglobulins, plasma exchange, and targeted biological agents) may help to manage symptoms, many patients still suffer from poor response, drug intolerance and disease relapse, highlighting a significant unmet clinical need. Telitacicept is the world's first approved innovative BLyS/APRIL dual-targeting fusion protein drug for MG. The pathology of MG is characterized by the autoantibodies produced by pathological B cells attacking the neuromuscular junction protein (such as acetylcholine receptor, muscle-specific tyrosine kinase). Telitacicept can block both BLyS and APRIL signaling pathways, effectively inhibiting abnormally activated B cells and reducing the production of pathogenic autoantibodies, which is expected to interfere with the source of disease progress of MG. Telitacicept was approved for marketing in China in May this year. Its phase III clinical trial boasts excellent results: after treatment with telitacicept for 24 weeks, improvement ≥ 3 points in myasthenia gravis-activities of daily living (MG-ADL) was observed in 98.1% of participants (12.0% in the placebo group) and improvements ≥ 5 points in quantitative myasthenia gravis (QMG) score was observed in 87% of participants (16.0% in the placebo group), with statistically significant difference in efficacy from the placebo group and manageable safety profile. The ODD granted by EMA represents the recognition of telitacicept's innovative mechanism of action and potential in treating MG. RemeGen is proceeding the global multi-center phase III clinical trial of telitacicept in patients with MG to bring the breakthrough treatment option to more patients globally. View original content to download multimedia: SOURCE RemeGen Co., Ltd 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
4 days ago
- Business
- Yahoo
CERo Therapeutics Holdings, Inc. Announces FDA Orphan Drug Designation Granted to CER-1236 for the Treatment of Acute Myeloid Leukemia (AML)
SOUTH SAN FRANSCISCO, Calif., June 17, 2025 (GLOBE NEWSWIRE) -- CERo Therapeutics Holdings, Inc., (Nasdaq: CERO) ('CERo' or the 'Company') an innovative immunotherapy company seeking to advance the next generation of engineered T cell therapeutics that deploy phagocytic mechanisms, announces that the U.S. Food and Drug Administration (FDA) has granted CERo's Orphan Drug Designation (ODD) for the company's lead drug candidate CER-1236, for the treatment of acute myeloid leukemia (AML). CER-1236 is an innovative therapy that engineers a cancer patient's own T cell therapeutics that deploy phagocytic (i.e., target-cell eating) mechanisms alongside the array of built-in target cell destroying mechanisms used by T cells. CER-1236 is currently in Phase 1 clinical trials for AML. The first-in-human, multi-center, open label, Phase 1/1b study is designed to evaluate the safety and preliminary efficacy of CER-1236 in patients with acute myeloid leukemia that is either relapsed/refractory, or in remission with measurable residual disease, or newly diagnosed patients with TP53 mutated MDS/AML or AML. The two-part study has begun with dose escalation to determine highest tolerated dose and recommended dose for Phase 2, followed by an expansion phase to evaluate safety and efficacy. Primary outcome measures include incidence of adverse events (AEs) and serious adverse events (SAEs), incidence of dose limited toxicities and estimation of overall response rate (ORR), complete response (CR), composite complete response (cCR), and measurable residual disease (MRD). Secondary outcome measures include pharmacokinetics (PK). Chris Ehrlich, CERo CEO, commented, 'Orphan Drug Designation underscores the importance of developing new treatments for AML, and the potential for CER-1236 to provide a new and differentiated approach toward treatment. We believe that we are at the forefront of innovation in immuno-oncology and are grateful for the recognition from FDA. We look forward to providing updates on our trial in the near term.' The FDA's Orphan Drug program is designed to advance the development of drugs that treat a condition affecting 200,000 or fewer US patients annually. ODD status is given to medicinal products that represent a significant benefit over existing treatments and are intended for the treatment of a disease that is life-threatening or chronically debilitating. The ODD designation qualifies CERo and CER-1236 for certain incentives, which include FDA assistance in designing clinical trials, access to the FDA Orphan Drug Grants Program, exemption from the drug approval application fee and eligibility for seven years of marketing exclusivity. About CERo Therapeutics Holdings, Inc. CERo is an innovative immunotherapy company advancing the development of next generation engineered T cell therapeutics for the treatment of cancer. Its proprietary approach to T cell engineering, which enables it to integrate certain desirable characteristics of both innate and adaptive immunity into a single therapeutic construct, is designed to engage the body's full immune repertoire to achieve optimized cancer therapy. This novel cellular immunotherapy platform is expected to redirect patient-derived T cells to eliminate tumors by building in engulfment pathways that employ phagocytic mechanisms to destroy cancer cells, creating what CERo refers to as Chimeric Engulfment Receptor T cells ('CER-T'). CERo believes the differentiated activity of CER-T cells will afford them greater therapeutic application than currently approved chimeric antigen receptor ('CAR-T') cell therapy, as the use of CER-T may potentially span both hematological malignancies and solid tumors. CERo has commenced clinical trials for its lead product candidate CER-1236 for hematological malignancies. Forward-Looking Statements This communication contains statements that are forward-looking and as such are not historical facts. This includes, without limitation, statements regarding the financial position, business strategy and the plans and objectives of management for future operations of CERo, as well as statements regarding the Company's plans to regain compliance with Nasdaq listing requirements and the ability for the Company's securities to remain listed on Nasdaq. These statements constitute projections, forecasts and forward-looking statements, and are not guarantees of performance. Such statements can be identified by the fact that they do not relate strictly to historical or current facts. When used in this communication, words such as 'anticipate,' 'believe,' 'continue,' 'could,' 'estimate,' 'expect,' 'intend,' 'may,' 'might,' 'plan,' 'possible,' 'potential,' 'predict,' 'project,' 'should,' 'strive,' 'would' and similar expressions may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. When CERo discusses its strategies or plans, it is making projections, forecasts or forward-looking statements. Such statements are based on the beliefs of, as well as assumptions made by and information currently available to, CERo's management. Actual results could differ from those implied by the forward-looking statements in this communication. Certain risks that could cause actual results to differ are set forth in CERo's filings with the Securities and Exchange Commission, including its most recent Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q, and the documents incorporated by reference therein. The risks described in CERo's filings with the Securities and Exchange Commission are not exhaustive. New risk factors emerge from time to time, and it is not possible to predict all such risk factors, nor can CERo assess the impact of all such risk factors on its 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. Forward-looking statements are not guarantees of performance. You should not put undue reliance on these statements, which speak only as of the date hereof. All forward-looking statements made by CERo or persons acting on its behalf are expressly qualified in their entirety by the foregoing cautionary statements. CERo undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Contact: Chris EhrlichChief Executive Officerchris@ Investors: CORE IRinvestors@
Yahoo
5 days ago
- Business
- Yahoo
Circle Pharma Receives FDA Orphan Drug Designation for CID-078 for the Treatment of Small Cell Lung Cancer
SOUTH SAN FRANCISCO, Calif., June 16, 2025--(BUSINESS WIRE)--Circle Pharma, a clinical-stage biopharmaceutical company advancing macrocycle therapeutics for difficult-to-treat cancers, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CID-078 for the treatment of small cell lung cancer (SCLC). Small-cell lung cancer is a highly aggressive form of lung cancer that accounts for approximately 13–15% of all lung cancer cases1 and is strongly linked to tobacco exposure. Despite existing treatments, SCLC has a high recurrence rate and is associated with poor overall prognosis. While improvements in overall survival are occurring with newer therapies, most patients experience rapid disease progression2. "The Orphan Drug Designation from the FDA underscores both the seriousness of small cell lung cancer and the lack of effective treatment options," said Michael C. Cox, PharmD, MHSc, BCOP, SVP, and head of early development Circle Pharma. "We are committed to accelerating the clinical development of CID-078 to offer new hope for patients who face limited therapeutic choices." The FDA's Orphan Drug Designation program is intended to promote the development of drugs for rare diseases or conditions affecting fewer than 200,000 people in the United States3. This designation provides several development incentives, including seven years of market exclusivity upon regulatory approval, tax credits for qualified clinical trial costs, and eligibility to apply for FDA-administered research grants4. Circle Pharma has initiated a Phase 1 clinical trial (NCT06577987) of CID-078 to evaluate its safety, tolerability, pharmacokinetics, and early signs of anti-tumor activity in patients with advanced solid tumors, including SCLC. About CID-078, Circle Pharma's Cyclin A/B RxL Inhibitor Program CID-078 is an orally bioavailable macrocycle with dual cyclin A and B RxL inhibitory activity that selectively targets tumor cells with oncogenic alterations that cause cell cycle dysregulation. In biochemical and cellular studies, Circle Pharma's cyclin A/B RxL inhibitors have been shown to potently and selectively disrupt the protein-to-protein interaction between cyclins A and B and their key substrates and modulators, including E2F (a substrate of cyclin A) and Myt1 (a modulator of cyclin B). Preclinical studies have demonstrated the ability of these cyclin A/B RxL inhibitors to cause single-agent tumor regressions in multiple in vivo models. A multi-center phase 1 clinical trial (NCT06577987) is currently enrolling patients. About Circle Pharma, Inc. South San Francisco-based Circle Pharma is a clinical-stage biopharmaceutical company harnessing the power of macrocycles to develop therapies for cancer and other serious illnesses. The company's proprietary MXMO™ platform overcomes key challenges in macrocycle drug development, enabling the creation of intrinsically cell-permeable and orally bioavailable therapies for historically undruggable targets. Circle Pharma's pipeline is focused on targeting cyclins, key regulators of the cell cycle that drive many cancers. Its lead program, CID-078, a cyclin A/B-RxL inhibitor, is in a Phase 1 clinical trial (NCT06577987) for patients with advanced solid tumors. To learn more about Circle Pharma, please visit American Cancer Society. What is Small Cell Lung Cancer? National Cancer Institute. Small Cell Lung Cancer Treatment (PDQ®)–Patient Version. U.S. Food and Drug Administration. Developing Products for Rare Diseases & Conditions. U.S. Food and Drug Administration. Benefits of Orphan Drug Designation. View source version on Contacts Media Contact: Roslyn PattersonPhone: 650.825.4099Email:
Business Upturn
5 days ago
- Business
- Business Upturn
Can-Fite to Present Phase IIa Pancreatic Cancer Study Progress During Partnering Meetings at the 2025 BIO International Convention in Boston
Ramat Gan, Israel, June 16, 2025 (GLOBE NEWSWIRE) — Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF), a clinical-stage biotechnology company developing a pipeline of proprietary small molecule drugs for the treatment of cancer and inflammatory diseases, today announced that Dr. Sari Fishman, Vice President of Business Development, will present an update on the Company's ongoing Phase IIa study in pancreatic cancer during partnering meetings at the 2025 BIO International Convention, taking place June 16–19 in Boston, MA (link). The Phase IIa clinical trial is open-label study evaluating Namodenoson in patients with advanced pancreatic adenocarcinoma whose disease has progressed following at least one prior line of therapy. The study is assessing the safety, clinical activity, and pharmacokinetics (PK) of Namodenoson, administered orally at a dose of 25 mg twice daily in continuous 28-day cycles. Approximately 20 evaluable patients are expected to be enrolled. The trial is led by Prof. Salomon Stemmer, a prominent Oncologist and Key Opinion Leader at the Davidoff Center, Rabin Medical Center, Israel. Namodenoson has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of pancreatic cancer. 'We are pleased to report that 50% of the planned patient cohort has already been enrolled and that Namodenoson has demonstrated a favourable safety profile,' stated Prof. Salomon Stemmer. 'There is a critical unmet need for safe and effective treatment options for patients with advanced pancreatic cancer who have exhausted standard therapies. This study gives us the opportunity to advance a novel therapeutic approach for this challenging disease, stated Dr. Sari Fishman, VP of Business Development at Can-Fite.' Can-Fite looks forward to engaging with potential partners and collaborators at BIO 2025 as it continues to progress its clinical pipeline. About Can-Fite BioPharma Ltd. Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF) is an advanced clinical stage drug development Company with a platform technology that is designed to address multi-billion dollar markets in the treatment of cancer, liver, and inflammatory disease. The Company's lead drug candidate, Piclidenoson recently reported topline results in a Phase 3 trial for psoriasis and commenced a pivotal Phase 3 trial. Can-Fite's liver drug, Namodenoson, is being evaluated in a Phase III trial for hepatocellular carcinoma (HCC), a Phase 2b trial for the treatment of MASH, and in a Phase 2a study in pancreatic cancer. Namodenoson has been granted Orphan Drug Designation in the U.S. and Europe and Fast Track Designation as a second line treatment for HCC by the U.S. Food and Drug Administration. Namodenoson has also shown proof of concept to potentially treat other cancers including colon, prostate, and melanoma. CF602, the Company's third drug candidate, has shown efficacy in the treatment of erectile dysfunction. These drugs have an excellent safety profile with experience in over 1,600 patients in clinical studies to date. For more information please visit: Forward-Looking Statements This press release may contain forward-looking statements, about Can-Fite's expectations, beliefs or intentions regarding, among other things, its product development efforts, business, financial condition, results of operations, strategies or prospects. All statements in this communication, other than those relating to historical facts, are 'forward looking statements'. Forward-looking statements can be identified by the use of forward-looking words such as 'believe,' 'expect,' 'intend,' 'plan,' 'may,' 'should' or 'anticipate' or their negatives or other variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical or current matters. For example, the Company is using forward-looking statements when it discusses the completion of the offerings, the satisfaction of customary closing conditions related to the offerings and the intended use of proceeds therefrom. Forward-looking statements relate to anticipated or expected events, activities, trends or results as of the date they are made. Because forward-looking statements relate to matters that have not yet occurred, these statements are inherently subject to known and unknown risks, uncertainties and other factors that may cause Can-Fite's actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Important factors that could cause actual results, performance or achievements to differ materially from those anticipated in these forward-looking statements include, among other things, our market and other conditions, history of losses and needs for additional capital to fund our operations and our inability to obtain additional capital on acceptable terms, or at all; uncertainties of cash flows and inability to meet working capital needs; the initiation, timing, progress and results of our preclinical studies, clinical trials and other product candidate development efforts; our ability to advance our product candidates into clinical trials or to successfully complete our preclinical studies or clinical trials; our receipt of regulatory approvals for our product candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of our product candidates; our ability to establish and maintain strategic partnerships and other corporate collaborations; the implementation of our business model and strategic plans for our business and product candidates; the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and our ability to operate our business without infringing the intellectual property rights of others; competitive companies, technologies and our industry; risks related to not satisfying the continued listing requirements of NYSE American; and statements as to the impact of the political and security situation in Israel on our business. More information on these risks, uncertainties and other factors is included from time to time in the 'Risk Factors' section of Can-Fite's Annual Report on Form 20-F filed with the SEC on April 14, 2025 and other public reports filed with the SEC and in its periodic filings with the TASE. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Can-Fite undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. Contact Can-Fite BioPharma Motti Farbstein [email protected] +972-3-9241114 Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. Business Upturn takes no editorial responsibility for the same. Ahmedabad Plane Crash
Associated Press
5 days ago
- Business
- Associated Press
Circle Pharma Receives FDA Orphan Drug Designation for CID-078 for the Treatment of Small Cell Lung Cancer
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Jun 16, 2025-- Circle Pharma, a clinical-stage biopharmaceutical company advancing macrocycle therapeutics for difficult-to-treat cancers, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CID-078 for the treatment of small cell lung cancer (SCLC). Small-cell lung cancer is a highly aggressive form of lung cancer that accounts for approximately 13–15% of all lung cancer cases 1 and is strongly linked to tobacco exposure. Despite existing treatments, SCLC has a high recurrence rate and is associated with poor overall prognosis. While improvements in overall survival are occurring with newer therapies, most patients experience rapid disease progression 2. 'The Orphan Drug Designation from the FDA underscores both the seriousness of small cell lung cancer and the lack of effective treatment options,' said Michael C. Cox, PharmD, MHSc, BCOP, SVP, and head of early development Circle Pharma. 'We are committed to accelerating the clinical development of CID-078 to offer new hope for patients who face limited therapeutic choices.' The FDA's Orphan Drug Designation program is intended to promote the development of drugs for rare diseases or conditions affecting fewer than 200,000 people in the United States 3. This designation provides several development incentives, including seven years of market exclusivity upon regulatory approval, tax credits for qualified clinical trial costs, and eligibility to apply for FDA-administered research grants 4. Circle Pharma has initiated a Phase 1 clinical trial ( NCT06577987 ) of CID-078 to evaluate its safety, tolerability, pharmacokinetics, and early signs of anti-tumor activity in patients with advanced solid tumors, including SCLC. About CID-078, Circle Pharma's Cyclin A/B RxL Inhibitor Program CID-078 is an orally bioavailable macrocycle with dual cyclin A and B RxL inhibitory activity that selectively targets tumor cells with oncogenic alterations that cause cell cycle dysregulation. In biochemical and cellular studies, Circle Pharma's cyclin A/B RxL inhibitors have been shown to potently and selectively disrupt the protein-to-protein interaction between cyclins A and B and their key substrates and modulators, including E2F (a substrate of cyclin A) and Myt1 (a modulator of cyclin B). Preclinical studies have demonstrated the ability of these cyclin A/B RxL inhibitors to cause single-agent tumor regressions in multiple in vivo models. A multi-center phase 1 clinical trial ( NCT06577987 ) is currently enrolling patients. About Circle Pharma, Inc. South San Francisco-based Circle Pharma is a clinical-stage biopharmaceutical company harnessing the power of macrocycles to develop therapies for cancer and other serious illnesses. The company's proprietary MXMO™ platform overcomes key challenges in macrocycle drug development, enabling the creation of intrinsically cell-permeable and orally bioavailable therapies for historically undruggable targets. Circle Pharma's pipeline is focused on targeting cyclins, key regulators of the cell cycle that drive many cancers. Its lead program, CID-078, a cyclin A/B-RxL inhibitor, is in a Phase 1 clinical trial ( NCT06577987 ) for patients with advanced solid tumors. To learn more about Circle Pharma, please visit View source version on CONTACT: Media Contact: Roslyn Patterson Phone: 650.825.4099 Email: [email protected] KEYWORD: UNITED STATES NORTH AMERICA CALIFORNIA INDUSTRY KEYWORD: ONCOLOGY PROFESSIONAL SERVICES HEALTH FDA VENTURE CAPITAL PHARMACEUTICAL BIOTECHNOLOGY SOURCE: Circle Pharma, Inc. Copyright Business Wire 2025. PUB: 06/16/2025 07:05 AM/DISC: 06/16/2025 07:05 AM
