Latest news with #TKI
Business Upturn
6 days ago
- Business
- Business Upturn
Clearside Biomedical Announces Plan to Explore Strategic Alternatives to Advance its Proprietary Suprachoroidal Space (SCS®) Delivery Platform and Promising Ophthalmology Pipeline
– Proven In-Office, Repeatable, Non-Surgical Procedure for the Targeted Delivery of a Wide Variety of Therapies for Serious Retinal Diseases – – CLS-AX Phase 3-Ready Asset with Global Investigator Support and FDA Alignment on Pivotal Trial Program in wet AMD – – Validated Delivery Platform with Five Commercial and Late-Stage Development Suprachoroidal Licensing Collaborations – ALPHARETTA, Ga., July 17, 2025 (GLOBE NEWSWIRE) — Clearside Biomedical, Inc. (Nasdaq: CLSD), a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®), today announced plans to explore a full range of strategic alternatives to advance its SCS platform and drug development pipeline to maximize stockholder value. The Company has retained Piper Sandler, a leading investment bank with substantial experience in the biotechnology industry, to support it with the strategic evaluation process. Strategic alternatives under consideration include the sale, license, monetization and/or divestiture of one or more of the Company's assets and technologies, collaboration, partnership, merger, acquisition, joint ventures, or other strategic transactions. 'We strongly believe that our proprietary suprachoroidal delivery platform provides an effective and reliable way to target challenging retinal diseases that need longer lasting treatments,' said George Lasezkay, PharmD, JD, President and Chief Executive Officer. 'Our SCS Microinjector® enables a proven in-office, repeatable, non-surgical procedure for the targeted delivery of a wide variety of therapies to the macula, retina, and choroid. Based on our retinal expertise and intellectual property, we delivered the first commercial product using our SCS platform, which is now approved and commercialized in the U.S., approved in Singapore and Australia, and is currently under regulatory review in China and Canada.' Dr. Lasezkay further stated, 'We have successfully produced positive and highly competitive Phase 2b clinical data for CLS-AX for the treatment of wet AMD. We are proud of the fact that the CLS-AX ODYSSEY Phase 2b trial is the only TKI clinical trial to date to achieve positive safety and efficacy results from multiple TKI dosing in wet AMD patients. Further, in February 2025, we conducted a successful end of Phase 2 meeting with the FDA through which we gained alignment on a Phase 3 development plan for CLS-AX in wet AMD. In addition, our clinical development collaboration partners continue to make progress in advancing their later-stage suprachoroidal clinical programs utilizing our SCS Microinjector in various ophthalmic disorders.' Dr. Lasezkay continued, 'Clearside's innovative and pioneering achievements demonstrating the safety, simplicity and effectiveness of delivering drugs to the suprachoroidal space are the direct result of the many years of significant contributions by the current Clearside team and many of our past employees. I am proud of and grateful for their hard work and dedicated commitment to improving the lives of patients suffering from the burden of disabling, blinding diseases.' 'Given the current unpredictable economic environment and challenging fundraising conditions in the biopharmaceutical industry, we are taking the necessary next steps to evaluate strategic alternatives for the Company. In order to facilitate this process and conserve cash, while continuing our support of the Company's SCS Microinjector® licensees, all Clearside employees, including the Chief Executive Officer, Chief Financial Officer, and Chief Medical Officer, will transition into consulting roles with the Company this week. In addition, we will pause all internal research and development programs during this process,' concluded Dr. Lasezkay. No agreement providing for any strategic transaction has been reached and there can be no assurance that this process will result in any such transaction. Clearside has not set a timetable for the strategic review process. Clearside does not intend to provide updates until the Board approves a specific action or otherwise determines that disclosure is appropriate or required. Company Highlights SCS Injection Platform: The SCS Microinjector is a proven in-office, repeatable, non-surgical procedure for the targeted delivery of a wide variety of therapies to the macula, retina, or choroid to potentially preserve and improve vision in patients with sight-threatening eye diseases. Clearside has formulation expertise in developing small molecule suspensions that can be delivered into the suprachoroidal space, and commercial scale manufacturing capability for the SCS Microinjector that includes ISO certification. The Company successfully navigated the U.S. Food & Drug Administration (FDA) drug/device regulatory pathway to obtain commercial approval for its first product, XIPERE ® (triamcinolone acetonide injectable suspension) for suprachoroidal use for the treatment of uveitic macular edema. (triamcinolone acetonide injectable suspension) for suprachoroidal use for the treatment of uveitic macular edema. A permanent CPT code is assigned in the U.S. for suprachoroidal injections permitting physicians to receive higher reimbursement for administering any drug into the SCS versus the reimbursement of the current practice of injecting drugs into the vitreous. Internal Pipeline – CLS-AX Program: CLS-AX (axitinib injectable suspension) is a proprietary suspension of axitinib for suprachoroidal injection. CLS-AX is being developed for the treatment of neovascular age-related macular degeneration (wet AMD). Axitinib is a tyrosine kinase inhibitor (TKI) that achieves pan-VEGF blockade, directly inhibiting VEGF receptors-1, -2, and -3 with high potency and specificity. Clearside believes this broad VEGF blockade may have efficacy advantages over existing retinal therapies by acting at a different level of the angiogenesis cascade and may benefit patients who sub-optimally respond to current, more narrowly focused anti-VEGF therapies. Age-related macular degeneration causes a progressive loss of central vision and is the most common cause of legal blindness in individuals over age 55. Wet AMD is generally caused by abnormal blood vessels that leak fluid or blood into the macula, the part of the retina responsible for central vision, and accounts for the majority of vision loss in patients with this disorder. Suprachoroidal injection of CLS-AX has demonstrated meaningful potential in Phase 1/2a and Phase 2b wet AMD clinical trials. These data strongly support the potential efficacy, safety and versatility of CLS-AX to treat this chronic disease. Clearside conducted a successful End-of-Phase 2 meeting with the FDA and aligned on the essential components of a Phase 3 program. The Phase 3 program is designed to maximize the commercial potential for CLS-AX with the potential to provide physicians and their patients with dosing flexibility similar to the current standard of care anti-VEGF biologics, but with the extended durability of a TKI. This combination of dosing flexibility and durability may improve outcomes and reduce the treatment burden for patients suffering from these potentially blinding retinal disorders, as well as their caregivers. Internal Pipeline – Preclinical Small Molecule Programs: Clearside is evaluating various small molecules for a range of retinal diseases with high unmet medical need. Clearside is evaluating preclinical data on two approaches targeting Geographic Atrophy (GA) – improving choroidal perfusion and modulating pro-inflammatory cells. GA is a progressive, late-stage form of dry age-related macular degeneration characterized by well-defined areas of retinal cell loss in the macula, leading to irreversible central vision impairment. Delivery of small molecules via suprachoroidal injection enables comprehensive drug coverage of both the retina and choroid, while also potentially minimizing systemic and anterior segment side effects. Clearside is evaluating preclinical data on the combination of a steroid plus a TKI (axitinib formulation) for the treatment of Diabetic Macula Edema (DME). DME is the most common cause of vision loss in individuals with diabetes, in which fluid accumulates in the central part of the retina causing swelling and vision distortion due to leaky blood vessels damaged by prolonged high blood sugar. External Licensing Agreements: Clearside strategically partners its SCS injection platform with companies utilizing other ophthalmic therapeutic agents including gene therapies and anti-tumor agents. Clearside's innovative SCS Microinjector is being used in commercial ophthalmic products and promising clinical development programs by Aura Biosciences, Bausch + Lomb, BioCryst Pharmaceuticals, REGENXBIO and its global partner AbbVie, and Arctic Vision and its commercial partner Santen. About Clearside Biomedical, Inc. Clearside Biomedical, Inc. is a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®) to improve patient outcomes. Clearside's SCS injection platform, utilizing the Company's patented SCS Microinjector®, enables an in-office, repeatable, non-surgical procedure for the targeted and compartmentalized delivery of a wide variety of therapies to the macula, retina, or choroid to potentially preserve and improve vision in patients with sight-threatening eye diseases. Clearside is developing its own pipeline of small molecule product candidates for administration via its SCS Microinjector. The Company's lead program, CLS-AX (axitinib injectable suspension), is in development for the treatment of neovascular age-related macular degeneration (wet AMD). Planning for a Phase 3 program is underway. In addition, Clearside is evaluating various small molecules for the potential long-acting treatment of geographic atrophy (GA). Clearside developed and gained approval for its first product, XIPERE® (triamcinolone acetonide injectable suspension) for suprachoroidal use, which is available in the U.S. through a commercial partner. Clearside also strategically partners its SCS injection platform with companies utilizing other ophthalmic therapeutic innovations. For more information, please visit or follow us on LinkedIn and X. Cautionary Note Regarding Forward-Looking Statements Any statements contained in this press release that do not describe historical facts may constitute forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as 'believe', 'expect', 'may', 'plan', 'potential', 'will', and similar expressions, and are based on Clearside's current beliefs and expectations. These forward-looking statements include statements regarding the clinical development of CLS-AX, including the planned Phase 3 trial design, the potential benefits of CLS-AX, Clearside's suprachoroidal delivery technology and Clearside's SCS Microinjector®, and Clearside's pursuit of strategic alternatives and the entry into or completion of any strategic alternative transaction. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements. Risks and uncertainties that may cause actual results to differ materially include uncertainties inherent in the conduct of clinical trials, Clearside's reliance on third parties over which it may not always have full control, Clearside's ability to raise additional capital, and other risks and uncertainties that are described in Clearside's Annual Report on Form 10-K for the year ended December 31, 2024, filed with the U.S. Securities and Exchange Commission (SEC) on March 27, 2025 and Clearside's other Periodic Reports filed with the SEC. Any forward-looking statements speak only as of the date of this press release and are based on information available to Clearside as of the date of this release, and Clearside assumes no obligation to, and does not intend to, update any forward-looking statements, whether as a result of new information, future events or otherwise. Investor and Media Contacts: Jenny Kobin Remy Bernarda [email protected]
Bangkok Post
11-07-2025
- Health
- Bangkok Post
Thailand produces its first targeted cancer drug
Thailand has produced its first locally developed targeted therapy pill for cancer treatment. Imcranib 100 is the result of the scientific vision and leadership of Her Royal Highness Princess Chulabhorn Krom Phra Srisavangavadhana. Targeted therapy is a modern cancer treatment approach that focuses on attacking specific cancer cells while sparing healthy cells, thereby reducing side effects commonly associated with traditional treatments such as chemotherapy, according to a statement from the Chulabhorn Royal Academy (CRA). 'This method also allows for more personalised treatment plans tailored to each patient's condition,' it said. Driven by concern for cancer patients with limited access to effective medicines, HRH Princess Chulabhorn established a pharmaceutical manufacturing facility under royal initiative at Phimanmas Residence in Sattahip District of Chon Buri in 2020. The plant, operating under the CRA, is Thailand's first cancer drug manufacturing site certified to international GMDP PIC/s standards. It was created to ensure domestic production capacity from research to industrial scale, aiming to reduce reliance on costly drug imports and strengthen national pharmaceutical expertise. Her Royal Highness personally oversaw the plant's progress, participating in inspections and scientific operations within the quality control laboratory. She led efforts to maintain global standards in drug development, conducting chemical and physical analyses of raw materials and finished tablets. The culmination of these efforts is Imcranib 100, a tablet containing 100 milligrammes of Imatinib, registered with the Food and Drug Administration (FDA) on May 20 this year. The medicine is a tyrosine kinase inhibitor (TKI) that precisely targets cancer cells, used to treat conditions such as chronic myeloid leukaemia (CML), Philadelphia chromosome-positive acute leukaemia, gastrointestinal stromal tumours (GIST), and rare skin cancers (DFSP), said the CRA. Treatment using Imcranib 100 is now available at Chulabhorn Hospital, significantly reducing treatment costs and increasing access to care for Thai patients, according to the CRA. 'This groundbreaking achievement not only eases the burden of cancer for Thai citizens but also enhances Thailand's capacity in pharmaceutical formulation, manufacturing, quality control, pharmacological testing, and regulatory processes,' the statement said. 'It lays the foundation for future cancer drug development and strengthens national drug security for sustainable public health improvement.' Additionally, the Chulabhorn Research Institute has successfully developed Thailand's first targeted biological drug, Trastuzumab (registered as Herdara), also approved by the FDA on May 20.
Yahoo
25-06-2025
- Business
- Yahoo
Nuvalent price target raised to $130 from $110 at H.C. Wainwright
H.C. Wainwright analyst Swayampakula Ramakanth raised the firm's price target on Nuvalent (NUVL) to $130 from $110 and keeps a Buy rating on the shares. The company reported positive topline results from the Phase 1/2 ARROS-1 study evaluating zidesamtinib to treat ROS1-positive non-small cell lung cancer patients that have received prior (tyrosine kinase inhibitor therapy, the analyst tells investors in a research note. The firm says the overall response rate among all patients is 44%, and the patients showed good durability with duration of responses of 78% at 12 months and 62% at 18 months. H.C. Wainwright believes the data was 'particularly encouraging' in the 55 patients who received only one prior line of TKI therapy, who achieved overall response rate of 51% and an 18- month duration of response of 93%. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See today's best-performing stocks on TipRanks >> Read More on NUVL: Disclaimer & DisclosureReport an Issue Nuvalent's Zidesamib: Promising Best-in-Class ROS1 TKI with Strong Efficacy and Safety Profile Nuvalent Announces Positive Data for Zidesamtinib Trial Nuvalent Announces New Director and Annual Meeting Results Nuvalent's Zidesamtinib: Promising Efficacy and Safety Drive Buy Rating Nuvalent Board Member Emily Conley Resigns 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
17-06-2025
- Business
- Yahoo
Enliven Therapeutics, Inc. (ELVN) Backed by Goldman Sachs After Strong Trial Data
Enliven Therapeutics, Inc. (NASDAQ:ELVN) is among the best NASDAQ stocks under $50 to buy. On Monday, analysts at Goldman Sachs started coverage on Enliven Therapeutics, Inc. (NASDAQ:ELVN) with a Buy rating and a $37.00 price target, implying a rise of about 60% from the current levels. The firm's confidence in the stock stems from favorable Phase 1 data for the company's chronic myeloid leukemia (CML) solution. The investment bank noted that ELVN-001's sustained efficacy data, presented during the European Hematology Association meeting, highlighted a 47% overall cumulative major molecular response (MMR) rate by 24 weeks and a 41% response in tyrosine kinase inhibitor (TKI)-resistant patients. These results surpassed the fourth-generation Scemblix by Novartis (SIX:NOVN) figures, with a difference of 10% and 32%, respectively. A biologist in a laboratory examining a microscope for small molecule inhibitors. The company is all set to launch a Phase 3 trial in 2026, which in itself is a $3 billion opportunity. If we look at the three-year return, Enliven Therapeutics, Inc. (NASDAQ:ELVN) exhibited a return of a whopping 1,980.18% in contrast to the 64.53% return by the market. Calling this simply an 'amazing' performance would be an understatement. Enliven Therapeutics, Inc. (NASDAQ:ELVN), headquartered in Boulder, Colorado, is a clinical-stage biopharmaceutical company. The company focuses on the recognition and development of small-molecule inhibitors to assist people living with cancer, improving survival and overall well-being. While we acknowledge the potential of ELVN 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.
Medscape
04-06-2025
- Health
- Medscape
Fast Five Quiz: B-Cell Acute Lymphoblastic Leukemia
Though more common in pediatric populations, B-cell acute lymphoblastic leukemia (B-ALL) affects both children and adults, and it has several genetic subtypes that significantly affect prognosis and treatment decisions. Despite advances in treatment protocols, resistance mechanisms remain a significant challenge. However, some genetic subtypes are associated with more favorable prognosis, and the nuances of this disease are evident in the cascading treatment algorithm recommended by the National Comprehensive Cancer Network (NCCN) for pediatric and adult cases. What do you know about B-ALL? Check your knowledge with this quick quiz. The treatment of B-ALL involves a complex and intensive series of steps. In the induction phase, glucocorticoids are typically used to reduce tumor burden by clearing leukemic cells from the bone marrow. However, point mutations in the NR3C1 gene generally cause resistance to glucocorticoid therapy, which can significantly affect prognosis. Mutations in the CREBBP gene also cause resistance to glucocorticoids DHFR , FPGS , and TYMS genes have been shown to generally cause resistance to methotrexate, not glucocorticoids. Learn more about corticosteroids in B-ALL. In risk stratification for B-ALL, the NCCN notes that having a white blood cell count of > 30 x 109/L is considered a high-risk feature. This is consistent with European guidelines. The NCCN also states that age over 35 years is another high-risk feature for B-ALL. ETP phenotype is a high-risk feature for T-cell acute lymphoblastic leukemia rather than the B-ALL subtype. Hyperdiploidy (a molecular subtype of B-ALL with 51-65 chromosomes) is considered standard risk by the NCCN; data have shown better prognosis for pediatric patients with this subtype. Learn more about risk stratification for B-ALL. TKIs are an important component of induction, consolidation, and maintenance treatment for Ph+ B-ALL. They are recommended by the NCCN for use in conjunction with other drugs, such as corticosteroids, blinatumomab, and inotuzumab ozogamicin (depending on treatment phase and disease severity) and as a treatment post-HCT. They recommend continuing TKI therapy for at least 2 years after HCT, although they note that the 'optimal duration' is unknown for this population. A recent review, however, notes that despite their success in patients with Ph+ B-ALL, approximately 25% of cases will develop resistance to TKIs. Given this possibility, the NCCN notes that clinicians should consider prior TKI intolerance, dose used, BCR::ABL1 mutations, and disease-related features when choosing a specific TKI; they specifically recommend bosutinib, dasatinib, imatinib, nilotinib, or ponatinib as options for TKI therapy. Learn more about Ph+ B-ALL treatments. The DUX4-rearranged subtype of B-ALL is considered to have a favorable prognosis in adolescents and young adults, compared with MEF2D-rearranged, CDX2/UBTF, and IDH1/2; these subtypes generally have inferior prognosis in the same populations (although MEF2D-rearranged has intermediate prognosis in adults). Specifically, DUX4-rearranged B-ALL has been associated with 93% event-free survival and overall survival in pediatric patients, and adolescent and young-adult patients also see longer disease-free survival after complete remission is achieved. Learn more about B-ALL genomics. Several genetic subtypes of B-ALL are associated with varying prognoses, and the outcomes differences seen between pediatric and adult patients can be partly explained by the different subtypes expressed by these populations. Further, new therapies have enabled high survival rates among pediatric patients with B-ALL, with long-term survival of up to 90% in this population. Prognosis for B-ALL in adolescents and adults is comparatively poor. For example, a population-based study reported the following declining survival rates with age: 74% for patients 15-19 years old, 59% for patients 20-39 years old, and 43% for those 40-59 years old. However, the researchers explained that these percentages still demonstrate improvements, as survival rates were overall lower in the 1980s and 1990s. Learn more about B-ALL prognosis.
