Latest news with #BTD
Yahoo
20-05-2025
- Business
- Yahoo
Viridian Therapeutics Announces Positive Long-Term Durability Data from the Veligrotug Phase 3 THRIVE Clinical Trial in Patients with Active Thyroid Eye Disease (TED)
- 70% of patients treated with veligrotug in THRIVE who were proptosis responders at week 15 maintained their response at week 52 - - Veligrotug recently received Breakthrough Therapy Designation (BTD), supporting eligibility for Priority Review; the BTD request was based on veligrotug's (i) consistent and robust improvement and resolution of diplopia in chronic TED, and (ii) rapid onset of proptosis response - - Biologics License Application (BLA) submission for veligrotug is on track for second half 2025 - - Actively preparing organization for planned U.S. commercial launch in 2026 - WALTHAM, Mass., May 20, 2025--(BUSINESS WIRE)--Viridian Therapeutics, Inc. (Nasdaq: VRDN), a biopharmaceutical company focused on discovering, developing, and commercializing potential best-in-class medicines for serious and rare diseases, today announced positive long-term durability data from the THRIVE phase 3 clinical trial of veligrotug ("veli"), an intravenously delivered anti-insulin-like growth factor-1 receptor (IGF-1R) antibody, in patients with active thyroid eye disease (TED). TED is an autoimmune condition characterized by inflammation, growth, and damage to tissues around and behind the eye. The THRIVE phase 3 clinical trial in active TED evaluated 5 infusions of veli or placebo every three weeks with primary topline analysis at week 15 and then followed patients through week 52. Positive Veligrotug Durability at 52 Weeks 70% of veligrotug patients (21/30) in THRIVE, who were proptosis responders at week 15 and continued follow-up to the end of the study at week 52, maintained their proptosis response. Maintenance of response is defined as responders at week 15 who still had at least a 2-millimeter (mm) reduction in proptosis compared to baseline at week 52, without worsening in the fellow eye (≥2 mm increase), as measured by exophthalmometry. There were no changes to the safety profile in the follow-up period. The vast majority of adverse events reported at the week 15 primary analysis had resolved by week 52. "We view the strength of today's durability and safety resolution data as reinforcing veli's strong and consistently robust clinical profile," said Steve Mahoney, Viridian's President and CEO. "We believe that the totality of veligrotug's clinical data continues to demonstrate its potential to be the treatment-of-choice for patients living with TED. We believe these data, together with a streamlined dosing regimen of five infusions, position veli to become a market leading TED therapeutic, if approved. We continue to make great progress towards submitting the BLA in the second half of this year and preparing for a potential launch in 2026." Robust Veligrotug Topline Clinical Profile for Active and Chronic TED As announced in late 2024, veligrotug met all of its primary and secondary endpoints and was generally well-tolerated in its pivotal phase 3 clinical trials, THRIVE and THRIVE-2, for active and chronic TED, respectively. Veligrotug demonstrated a rapid onset of treatment effect and statistically significant and clinically meaningful reduction and resolution of diplopia in both clinical trials. THRIVE-2 was the first data set from a global phase 3 clinical trial in chronic TED patients to demonstrate statistically significant diplopia response and resolution. Together, THRIVE and THRIVE-2 comprise the largest pivotal program to date in TED. About Veligrotug Veligrotug is an intravenously (IV) delivered, anti-insulin-like growth factor-1 receptor (IGF-1R) antibody in phase 3 development for thyroid eye disease, with the potential to be the IV treatment-of-choice for active and chronic TED patients. IGF-1R is a clinically and commercially validated target for thyroid eye disease (TED) with U.S. revenues of approximately $2 billion in 2024. Veligrotug has the potential to improve patient experience with a differentiated dosing regimen that features a shorter infusion time and fewer infusions compared to the currently approved and marketed IGF-1R inhibitor. In its pivotal phase 3 clinical trials, THRIVE and THRIVE-2, veligrotug met all of its primary and secondary endpoints. Veligrotug demonstrated a rapid onset of treatment effect and statistically significant and clinically meaningful reduction and resolution of diplopia in both clinical trials. THRIVE-2 was the first demonstration in a global phase 3 clinical trial of a statistically significant diplopia response and resolution in chronic TED patients. Veligrotug was generally well tolerated. Viridian believes that the robust veligrotug clinical profile has the potential to establish a strong position in the TED commercial market, if approved, and may help facilitate the introduction of VRDN-003, its potential best-in-class subcutaneous IGF-1R antibody for TED. About Viridian Therapeutics Viridian is a biopharmaceutical company focused on discovering, developing and commercializing potential best-in-class medicines for patients with serious and rare diseases. Viridian's expertise in antibody discovery and protein engineering enables the development of differentiated therapeutic candidates for previously validated drug targets in commercially established disease areas. Viridian is advancing multiple candidates in the clinic for the treatment of patients with thyroid eye disease (TED). The company is conducting a pivotal program for veligrotug (VRDN-001), including two global phase 3 clinical trials (THRIVE and THRIVE-2), to evaluate its efficacy and safety in patients with active and chronic TED. Both THRIVE and THRIVE-2 reported positive topline data, meeting all the primary and secondary endpoints of each study. Viridian is also advancing VRDN-003 as a potential best-in-class subcutaneous therapy for the treatment of TED, including two ongoing global phase 3 pivotal clinical trials, REVEAL-1 and REVEAL-2, to evaluate the efficacy and safety of VRDN-003 in patients with active and chronic TED. In addition to its TED portfolio, Viridian is advancing a novel portfolio of neonatal Fc receptor (FcRn) inhibitors, including VRDN-006 and VRDN-008, which has the potential to be developed in multiple autoimmune diseases. Viridian is based in Waltham, Massachusetts. For more information, please visit Follow Viridian on LinkedIn and X. Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of words such as, but not limited to, "anticipate," "believe," "become," "continue," "could," "design," "estimate," "expect," "intend," "may," "might," "on track," "plan," "potential," "predict," "project," "should," "target," "will," or "would" or other similar terms or expressions that concern our expectations, plans and intentions. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations, and assumptions. Forward-looking statements include, without limitation, statements regarding: clinical development and anticipated commercialization of Viridian's product candidates, including veligrotug (formerly VRDN-001) and VRDN-003; the potential utility, efficacy, potency, safety, clinical benefits, clinical response, convenience, and number of indications of veligrotug and VRDN-003, including Viridian's view of the strength of the THRIVE durability and safety resolution data and veligrotug's robust clinical profile; veligrotug's potential to be the IV treatment-of-choice for active and chronic TED; the impact of Breakthrough Therapy Designation, including eligibility for Priority Review, or any other FDA designations; regulatory interactions and anticipated timing of regulatory submissions, including the anticipated BLA submission for veligrotug in the second half of 2025; potential market sizes and market opportunities for Viridian's product candidates, including Viridian's belief that veligrotug is positioned to become a market leading TED therapeutic, if approved; Viridian's product candidates potentially being best-in-class; whether veligrotug will serve an unmet need; veligrotug's potential to improve patient experience over existing therapies; and Viridian's expectations regarding the potential commercialization of veligrotug and VRDN-003, if approved, including the potential U.S. launch of veligrotug in 2026. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Such forward-looking statements are subject to a number of material risks and uncertainties including but not limited to: potential utility, efficacy, potency, safety, clinical benefits, clinical response, and convenience of Viridian's product candidates; that results or data from completed or ongoing clinical trials may not be representative of the results of ongoing or future clinical trials; that preliminary data may not be representative of final data; expectations and changes regarding the timing for regulatory filings; regulatory interactions; uncertainty and potential delays related to clinical drug development; the timing of and our ability to obtain and maintain regulatory approvals for our therapeutic candidates; competition from other therapies or products; estimates of market size; our future operating results and financial performance; Viridian's intellectual property position; that our product candidates may not be commercially successful, if approved; and other risks described from time to time in the "Risk Factors" section of our filings with the Securities and Exchange Commission (SEC), including those described in our most recent Annual Report on Form 10-K or Quarterly Report on Form 10-Q, as applicable, and supplemented from time to time by our Current Reports on Form 8-K. Any forward-looking statement speaks only as of the date on which it was made. Neither the company, nor its affiliates, advisors, or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing the company's views as of any date subsequent to the date hereof. View source version on Contacts IR@ Media@ 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
12-05-2025
- Business
- Yahoo
InnoCare's Mesutoclax Receives Breakthrough Therapy Designation from China's NMPA
BEIJING, May 12, 2025--(BUSINESS WIRE)--InnoCare Pharma (HKEX: 09969; SSE: 688428), a leading biopharmaceutical company focusing on the treatment of cancer and autoimmune diseases, announced today that its B-cell lymphoma-2 (BCL2) inhibitor, Mesutoclax (ICP-248), has been granted Breakthrough Therapy Designation (BTD) by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) for the treatment of BTKi-treated relapsed or refractory mantle cell lymphoma (R/R MCL). This marks the first BCL2 inhibitor to receive BTD recognition in China! Mesutoclax is a novel, orally bioavailable BCL2 selective inhibitor, developed as monotherapy or in combination with orelabrutinib for the treatment of chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL), mantle cell lymphoma (MCL), and other non-Hodgkin's lymphomas (NHLs), and as well as acute myeloid leukemia (AML). BCL2 is an important regulatory protein in the apoptosis pathway, and its abnormal expression is associated with the development of various hematologic malignancies. Mesutoclax exerts anti-tumor activity by selectively inhibiting BCL2 and restoring the normal apoptosis process in cancer cells. Dr. Jasmine Cui, the Co-founder, Chairwoman, and CEO of InnoCare, said, "We are delighted that mesutoclax has been granted Breakthrough Therapy Designation, which will help expedite multi-center, multi-indication clinical trials for mesutoclax in China and globally to benefit patients as early as possible." Clinical trials of mesutoclax are ongoing both in China and globally, including a Phase III registrational trial of mesutoclax in combination with orelabrutinib as a first line therapy for the treatment of CLL/SLL patients, as well as a clinical trial of mesutoclax for the treatment of AML. The Breakthrough Therapy Designation (BTD) by the CDE aims to accelerate the clinical development of new drugs that demonstrate significant clinical advantages. New drugs granted BTD are typically intended for diseases that are life-threatening or severely impair quality of life, and have demonstrated clear advantages in efficacy or safety during clinical trials. About InnoCare InnoCare is a commercial stage biopharmaceutical company committed to discovering, developing, and commercializing first-in-class and/or best-in-class drugs for the treatment of cancers and autoimmune diseases with unmet medical needs in China and worldwide. InnoCare has branches in Beijing, Nanjing, Shanghai, Guangzhou, Hong Kong, and the United States. InnoCare Forward-looking Statements This report contains the disclosure of some forward-looking statements. Except for statements of facts, all other statements can be regarded as forward-looking statements, that is, about our or our management's intentions, plans, beliefs, or expectations that will or may occur in the future. Such statements are assumptions and estimates made by our management based on its experience and knowledge of historical trends, current conditions, expected future development and other related factors. This forward-looking statement does not guarantee future performance, and actual results, development and business decisions may not match the expectations of the forward-looking statement. Our forward-looking statements are also subject to a large number of risks and uncertainties, which may affect our short-term and long-term performance. View source version on Contacts Media Chunhua Investors 86-10-66609999ir@ 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
Business Wire
12-05-2025
- Business
- Business Wire
InnoCare's Mesutoclax Receives Breakthrough Therapy Designation from China's NMPA
BEIJING--(BUSINESS WIRE)--InnoCare Pharma (HKEX: 09969; SSE: 688428), a leading biopharmaceutical company focusing on the treatment of cancer and autoimmune diseases, announced today that its B-cell lymphoma-2 (BCL2) inhibitor, Mesutoclax (ICP-248), has been granted Breakthrough Therapy Designation (BTD) by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) for the treatment of BTKi-treated relapsed or refractory mantle cell lymphoma (R/R MCL). This marks the first BCL2 inhibitor to receive BTD recognition in China! This marks the first BCL2 inhibitor to receive BTD recognition in China! Clinical trials of mesutoclax are ongoing both in China and globally, including a Phase III registrational trial for CLL/SLL and a clinical trial for AML. Share Mesutoclax is a novel, orally bioavailable BCL2 selective inhibitor, developed as monotherapy or in combination with orelabrutinib for the treatment of chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL), mantle cell lymphoma (MCL), and other non-Hodgkin's lymphomas (NHLs), and as well as acute myeloid leukemia (AML). BCL2 is an important regulatory protein in the apoptosis pathway, and its abnormal expression is associated with the development of various hematologic malignancies. Mesutoclax exerts anti-tumor activity by selectively inhibiting BCL2 and restoring the normal apoptosis process in cancer cells. Dr. Jasmine Cui, the Co-founder, Chairwoman, and CEO of InnoCare, said, 'We are delighted that mesutoclax has been granted Breakthrough Therapy Designation, which will help expedite multi-center, multi-indication clinical trials for mesutoclax in China and globally to benefit patients as early as possible.' Clinical trials of mesutoclax are ongoing both in China and globally, including a Phase III registrational trial of mesutoclax in combination with orelabrutinib as a first line therapy for the treatment of CLL/SLL patients, as well as a clinical trial of mesutoclax for the treatment of AML. The Breakthrough Therapy Designation (BTD) by the CDE aims to accelerate the clinical development of new drugs that demonstrate significant clinical advantages. New drugs granted BTD are typically intended for diseases that are life-threatening or severely impair quality of life, and have demonstrated clear advantages in efficacy or safety during clinical trials. About InnoCare InnoCare is a commercial stage biopharmaceutical company committed to discovering, developing, and commercializing first-in-class and/or best-in-class drugs for the treatment of cancers and autoimmune diseases with unmet medical needs in China and worldwide. InnoCare has branches in Beijing, Nanjing, Shanghai, Guangzhou, Hong Kong, and the United States. InnoCare Forward-looking Statements This report contains the disclosure of some forward-looking statements. Except for statements of facts, all other statements can be regarded as forward-looking statements, that is, about our or our management's intentions, plans, beliefs, or expectations that will or may occur in the future. Such statements are assumptions and estimates made by our management based on its experience and knowledge of historical trends, current conditions, expected future development and other related factors. This forward-looking statement does not guarantee future performance, and actual results, development and business decisions may not match the expectations of the forward-looking statement. Our forward-looking statements are also subject to a large number of risks and uncertainties, which may affect our short-term and long-term performance.
Business Wire
22-04-2025
- Business
- Business Wire
FDA Grants Breakthrough Therapy Designation for BrainChild Bio's B7-H3 CAR T-Cell Therapy for Incurable Pediatric Brain Tumors
SEATTLE & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- BrainChild Bio, Inc., a clinical-stage biotechnology company developing CAR T-cell therapies to treat tumors in the central nervous system (CNS), today announced that the investigational B7-H3 targeting autologous CAR T-cell therapy has been granted Breakthrough Therapy designation (BTD) by the U.S Food and Drug Administration (FDA) for the treatment of diffuse intrinsic pontine glioma (DIPG), an incurable pediatric brain tumor. This FDA designation was based on the promising overall survival benefit in patients with brain tumors treated with an autologous B7-H3 CAR T-cell therapy observed in the BrainChild-03 Phase 1 trial (NCT04185038), conducted by BrainChild Bio's academic partner, Seattle Children's, and recently published in Nature Medicine. 'Breakthrough Therapy designation gives us the possibility to accelerate the development path for BCB-276 as a CAR T-cell therapy that can potentially transform the treatment of DIPG,' stated Michael Jensen, MD, Founder and Chief Scientific Officer of BrainChild Bio. 'This designation is a major milestone for the children and families afflicted with these devastating brain tumors and represents a new paradigm for treating CNS brain tumors in children and adults, including a large number of patients suffering with glioblastomas and brain metastases.' FDA grants Breakthrough Therapy designation to investigational medicines that demonstrate the potential to treat a serious or life-threatening condition and show preliminary clinical evidence that the drug may show substantial clinical improvement over available therapies. Investigational medicines with BTD are provided early and more frequent interactions with the FDA to discuss the product candidate's development plan in addition to being eligible for rolling submission and priority review of the marketing application. 'This designation is an important milestone for Seattle Children's and demonstrates our continued momentum in pediatric brain cancer research,' said Dr. Jeff Sperring, Chief Executive Officer of Seattle Children's. 'We harness the power of research to bring potential cures to kids faster, and we're excited by the early promise shown by our work with BrainChild Bio to advance a potential CAR T therapy.' BrainChild Bio is preparing to advance BCB-276 in a Phase 2 multi-center, pivotal registration trial to support a potential Biologics License Application (BLA) to the FDA for the treatment of children and young adults with DIPG. This clinical plan is based on alignment between BrainChild Bio and FDA at a Type B meeting in late 2024. About Diffuse Intrinsic Pontine Glioma (DIPG) and Application of CAR T-cell Therapies Diffuse intrinsic pontine glioma (DIPG) is a primary high-grade brain tumor that arises in the pons and is uniformly fatal. DIPG affects approximately 300 children per year in the U.S. with the majority of diagnoses made in children between 5 and 10 years of age. Current standard-of-care treatment remains limited to palliative focal radiation therapy which results in a median overall survival of only about 11 months from diagnosis. 1 Barriers to effective therapies for DIPG include the precarious location of the tumor in the brainstem, the infiltrative growth of the tumor throughout normal brainstem functional anatomy, and the blood brain barrier that remains relatively intact during tumor progression preventing therapies from gaining access to the cancer. The barriers to effective therapies for DIPG can be effectively overcome by the locoregional delivery of appropriately targeted CAR T-cells directly into the cerebrospinal fluid via intracerebroventricular (ICV) dosing with an indwelling reservoir-catheter device. This enables the potential for extensive exposure of the pons to cerebrospinal fluid flow from the ventricular system, thus permitting infused CAR T-cells to directly access the tumor bed. This also allows for repetitive infusions of CAR T-cells to replenish the tumor bed, offering the potential for more durable and sustained efficacy. Additionally, with the blood brain barrier intact, this therapeutic approach can also minimize any on-target, off-tumor toxicities resulting from systemic exposure of CAR T cells. About BrainChild Bio BrainChild Bio, Inc., is a kids-first, clinical-stage biotechnology company harnessing the power of CAR T-cell technology to treat tumors in the central nervous system, prioritizing pediatric indications with plans to expand into adult CNS tumors, specifically Glioblastoma and brain metastasis. BrainChild Bio was launched out of Seattle Children's Therapeutics program and founded on the work of Dr. Michael Jensen, a pioneer in the cancer immunotherapy field and previous Chief Therapeutics Officer at Seattle Children's. BrainChild Bio is advancing a next-generation CAR T-cell therapy platform for tumors of the CNS that weaves together synthetic technologies, including multiplex targeting and enhanced potency controls, to enable multiple targets in a single CAR T-cell therapy, novel transgenes to increase potency, delivery technology for durable efficacy, and streamlined CAR T-cell design and manufacturing. BrainChild Bio's lead drug candidate is BCB-276, an autologous CAR T-cell therapy that targets the immune checkpoint B7-H3, that is advancing in clinical trials for the treatment of diffuse intrinsic pontine glioma (DIPG), a pediatric cancer that forms in the brainstem which currently has no approved treatments. More information is available at
