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Business Wire
06-08-2025
- Business
- Business Wire
Viridian Therapeutics Highlights Recent Progress and Reports Second Quarter 2025 Financial Results
WALTHAM, Mass.--(BUSINESS WIRE)--Viridian Therapeutics, Inc. (Nasdaq: VRDN), a biotechnology company focused on discovering, developing and commercializing potential best-in-class medicines for serious and rare diseases, today reported recent business highlights and financial results for the second quarter ended June 30, 2025. 'Veligrotug's recent Breakthrough Therapy Designation as well as the continued and consistent performance of veligrotug across all of the endpoints and timepoints in our pivotal clinical trials, including the latest update on durability of response, showcase the momentum Viridian is building as we approach our planned BLA filing and expected commercial launch,' said Steve Mahoney, Viridian's President and CEO. 'We are making extraordinary progress on our commercial preparation and we plan to be launch-ready on a Priority Review designation timeline, if we receive it. In parallel to U.S. commercial launch planning, the recently announced license agreement with Kissei to develop and commercialize veligrotug and VRDN-003 in Japan further validates the value of our TED programs and the potential broad global opportunities in front of us. Overall, we are very pleased with our progress across our portfolio, including continuing to advance our FcRn inhibitors, VRDN-006 and VRDN-008, and look forward to sharing the outcomes from our upcoming milestones.' Recent Business Highlights Veligrotug for Active and Chronic Thyroid Eye Disease Positive Pivotal Trial Readouts. Veligrotug achieved all primary and secondary endpoints across proptosis, Clinical Activity Score (CAS), and diplopia in each of its two pivotal phase 3 clinical trials, THRIVE and THRIVE-2 for patients with active and chronic TED respectively. Robust Clinical Profile. This is the first and only drug candidate in chronic TED to demonstrate statistically significant and clinically meaningful improvement and resolution of diplopia in a global phase 3 clinical trial to date. Generally Well Tolerated Safety Profile. Veligrotug was generally well-tolerated in its pivotal phase 3 clinical trials, THRIVE and THRIVE-2. Breakthrough Therapy Designation. The FDA granted Breakthrough Therapy Designation (BTD) to veligrotug for the treatment of TED. Our application was based on veligrotug's (i) consistent and robust improvement and resolution of diplopia in chronic TED, and (ii) rapid onset of proptosis response. BTD supports eligibility for Priority Review which, if received, could accelerate our timing for veli commercial launch. Durability of Response. Demonstrated positive long-term durability data in THRIVE, with 70% of proptosis responders at 15 weeks maintaining that response at 52 weeks, which is 40 weeks after patients received their last dose in the clinical trial. Biologics License Application. Viridian is on track to submit a BLA to the U.S. FDA in the second half of 2025 and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in the first half of 2026. VRDN-003 for Active and Chronic Thyroid Eye Disease Topline Data – Pivotal Clinical Trials. Anticipate topline data from both REVEAL-1 and REVEAL-2 in the first half of 2026; BLA submission planned for year-end 2026. Planned Commercial Profile. Plan to launch VRDN-003, if approved, with a commercially validated, low-volume autoinjector designed for patients to infrequently self-administer at home. Japan Licensing with Kissei Pharmaceutical As announced in July, Viridian entered into an exclusive license agreement with Kissei Pharmaceutical to develop and commercialize veligrotug and VRDN-003 in Japan. Viridian will receive an upfront cash payment of $70 million, with the potential to receive an additional $315 million in development, regulatory, and commercial milestones. Viridian also receives tiered royalties on net sales in Japan with percentages ranging from the 20s to mid-30s. Kissei will be responsible for all development, regulatory, and commercialization activities, and associated costs, in Japan. FcRn Inhibitor Portfolio VRDN-006 Healthy Volunteer Data. Viridian expects data from the VRDN-006 phase 1 clinical trial in healthy volunteers in Q3 2025, including proof-of-concept IgG reduction VRDN-008 Investigational New Drug (IND). Submission on track for year-end 2025 As previously disclosed, after a single, high dose in non-human primates, VRDN-008 showed a longer half-life head-to-head versus efgartigimod and a more sustained IgG reduction. Expected Upcoming Milestones Veligrotug BLA submission in second half 2025 U.S. commercial launch in 2026, if approved MAA submission in first half 2026 VRDN-003 Topline data in first half 2026 VRDN-006 Healthy volunteer clinical data in Q3 2025 VRDN-008 IND submission year-end 2025 Financial Results Cash Position: Cash, cash equivalents, and short-term investments were $563.4 million as of June 30, 2025, compared with $636.6 million as of March 31, 2025. The company believes that its current cash, cash equivalents, and short-term investments will be sufficient to fund its currently planned operations into the second half of 2027. R&D Expenses: Research and development expenses were $86.6 million during the three months ended June 30, 2025, compared to $56.2 million during the three months ended June 30, 2024. The increase in research and development expenses was driven by increased costs associated with conducting more clinical trials than the same period last year, including multiple ongoing phase 3 clinical trials for both veligrotug and VRDN-003 and a phase 1 clinical trial for VRDN-006, as well as increased personnel-related costs as a result of increased headcount. G&A Expenses: General and administrative expenses were $20.2 million during the three months ended June 30, 2025, compared to $16.1 million during the three months ended June 30, 2024. The increase was driven by increased costs associated with preparatory commercial activities for veligrotug, as well as increased legal, accounting, and other professional service costs and personnel-related costs to support the growing organization. Shares Outstanding: As of June 30, 2025, Viridian had 100,320,386 shares of common stock outstanding on an as-converted basis, which included 81,651,186 shares of common stock and an aggregate 18,669,200 shares of common stock issuable upon the conversion of 134,864 and 145,160 shares of Series A and Series B preferred stock, respectively. 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) and a portfolio of inhibitors to the neonatal Fc receptor (FcRn). In TED, the company is conducting a pivotal program for veligrotug, 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. For the FcRn portfolio, Viridian is developing VRDN-006, a Fc fragment that inhibits FcRn, and VRDN-008, a half-life extended FcRn inhibitor. About Veligrotug Veligrotug is an intravenously 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. Based on clinical data to date, veligrotug has demonstrated robust clinical activity and was generally well-tolerated. In its pivotal phase 3 clinical trials, THRIVE and THRIVE-2, veligrotug demonstrated a rapid onset of treatment effect and statistically significant and clinically meaningful reduction and resolution of diplopia. Both THRIVE and THRIVE-2 reported positive topline data, meeting all the primary and secondary endpoints of each study. This is the first data set from a global phase 3 clinical trial in chronic TED patients to demonstrate statistically significant diplopia response and resolution. About VRDN-003 VRDN-003 is a subcutaneously delivered, half-life extended, potential best-in-class anti-IGF-1R antibody. VRDN-003 has the same binding domain as veligrotug and was engineered to have a longer half-life. In a phase 1 healthy volunteer clinical trial, VRDN-003 showed a half-life of 40-50 days, 4-5x that of veligrotug. Pharmacokinetics modeling predicted that VRDN-003 exposure levels after Q4W and Q8W dosing achieve the range of veligrotug exposures that showed robust clinical activity in a two-infusion phase 2 clinical trial in TED. Viridian is conducting a pivotal program for VRDN-003, including two phase 3 clinical trials assessing VRDN-003 dosed Q4W and Q8W in active and chronic TED, REVEAL-1 and REVEAL-2, respectively. About VRDN-006 and VRDN-008 VRDN-006 is a highly selective Fc fragment which inhibits FcRn and is designed to be a convenient subcutaneous and self-administered option for patients. Viridian is studying VRDN-006 in a first-in-human phase 1 clinical trial in healthy volunteers. VRDN-008 is a half-life extended FcRn inhibitor comprising an Fc fragment and an albumin-binding domain designed to prolong IgG suppression and provide a potentially best-in-class subcutaneous option for patients. VRDN-008 showed a longer half-life than efgartigimod and led to a more sustained IgG reduction after a single, high dose in non-human primates. 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: preclinical development, clinical development, and anticipated commercialization of Viridian's product candidates veligrotug (formerly VRDN-001), VRDN-003, VRDN-006, and VRDN-008; anticipated data results and timing of their disclosure, including VRDN-003 topline data from the REVEAL-1 and REVEAL-2 trials in the first half of 2026 and anticipated VRDN-006 clinical data, including proof-of-concept IgG reduction data, in the third quarter of 2025; regulatory interactions and anticipated timing of regulatory submissions, including the anticipated BLA submissions for veligrotug in the second half of 2025 and VRDN-003 by year-end 2026, MAA submission for veligrotug in the first half of 2026, and IND submission for VRDN-008 by year-end 2025, pending data; the impact of Breakthrough Therapy Designation, including eligibility for Priority Review, or any other FDA designations; the potential utility, efficacy, potency, safety, clinical benefits, clinical response, convenience, and number of indications of veligrotug, VRDN-003, VRDN-006, and VRDN-008, 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; potential market sizes and market opportunities, including for Viridian's product candidates; Viridian's product candidates potentially being best-in-class; Viridian's expectations regarding the potential commercialization of veligrotug and VRDN-003, if approved, including the anticipated U.S. launch of veligrotug in 2026, plans to launch VRDN-003 with a low-volume autoinjector, and in Japan under the agreement with Kissei; Viridian's partnership with Kissei, including that it supports the potential for broad global opportunities; Viridian's ability to receive development, regulatory, and commercial milestone payments and receive royalties on the commercial sale of our product candidates, if approved, pursuant to the agreement with Kissei; and that Viridian's cash, cash equivalents and short-term investments will be sufficient to fund its operations into the second half of 2027. 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; the timing, progress and plans for our ongoing or future research, preclinical, and clinical development programs; changes to trial protocols for ongoing or new clinical trials; expectations and changes regarding the timing for regulatory filings; regulatory interactions; expectations and changes regarding the timing for enrollment and data; uncertainty and potential delays related to clinical drug development; the duration and impact of regulatory delays in our clinical programs; the timing of and our ability to obtain and maintain regulatory approvals for our therapeutic candidates; manufacturing risks; competition from other therapies or products; estimates of market size; other matters that could affect the sufficiency of existing cash, cash equivalents, and short-term investments to fund operations; our financial position and projected cash runway; our future operating results and financial performance; Viridian's intellectual property position; the timing of preclinical and clinical trial activities and reporting results from same; 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.
Yahoo
10-06-2025
- Business
- Yahoo
China's Center for Drug Evaluation Accepts Merck KGaA, Darmstadt, Germany's Application for Marketing Authorization of Pimicotinib for Treatment of Tenosynovial Giant Cell Tumor
First global filing for potentially best-in-class TGCT treatment based on strong positive data from Phase 3 MANEUVER study Potential for pimicotinib to be first systemic TGCT therapy approved in China, following recent granting of Priority Review by CDE Additional applications planned in the US and other markets around the world Not intended for UK-based media DARMSTADT, Germany, June 10, 2025--(BUSINESS WIRE)--Merck KGaA, Darmstadt, Germany, a leading science and technology company, today announced that the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) officially accepted the company's application for marketing authorization of pimicotinib as a Class 1 innovative drug for adult patients with tenosynovial giant cell tumors (TGCT) requiring systemic treatment. The submission follows the granting of Priority Review to pimicotinib by the CDE in May for the treatment of patients with TGCT who require systemic therapy, which is expected to expedite the review process. Pimicotinib, a potentially best-in-class investigational colony stimulating factor-1 receptor (CSF-1R) inhibitor in development by Abbisko Therapeutics Co., Ltd., also has been granted breakthrough therapy designation (BTD) by the NMPA. "With the acceptance of our application for pimicotinib and the initiation of the priority review, we aim to offer patients in China the first approved systemic therapy for TGCT, addressing a tremendous unmet need in this country," said Hong Chow, Head of China and International, Healthcare business of Merck KGaA, Darmstadt, Germany. "Pimicotinib has demonstrated the ability to not only shrink tumors that affect their joints but also improve outcomes like mobility, pain and stiffness, highlighting its potential to be a best-in-class treatment for TGCT. In parallel, we are working to file a New Drug Application to the US Food and Drug Administration, with additional filings planned in other markets." TGCT is a locally aggressive and often recurring tumor of the joints that can cause high morbidity associated with swelling, pain, stiffness, and limited mobility of the affected joints, significantly impacting daily activities and quality of life in the primarily working-age population that it affects. If left untreated or in recurrent cases, TGCT can result in irreversible damage to the bone, joint and surrounding tissues. This highlights the need for well-tolerated and effective systemic treatments that can impact tumor growth while relieving the symptoms of the disease. The application is based on results from Part 1 of the global Phase 3 MANEUVER study, in which once-daily pimicotinib demonstrated a statistically significant improvement in the primary endpoint of objective response rate (ORR) assessed by blinded independent review committee (BIRC) compared with placebo at week 25 (54.0% vs. 3.2% for placebo; p<0.0001). The study also demonstrated statistically significant and clinically meaningful improvements in all secondary endpoints related to key patient-reported outcomes in TGCT, including improvements in active range of motion and physical function and reductions in stiffness and pain. The data were presented earlier this month at the 2025 ASCO Annual Meeting. About MANEUVERThe pivotal Phase 3 MANEUVER study is a three-part, randomized, double-blind, placebo-controlled study to assess the efficacy and safety of pimicotinib in patients with TGCT who require systemic therapy and have not received prior anti-CSF-1/CSF-1R therapy. The study is being conducted by Abbisko Therapeutics in China (n=45), Europe (n=28), and the US and Canada (n=21). In the double-blind Part 1, 94 patients were randomized 2:1 to receive either 50 mg QD of pimicotinib (n=63) or placebo (n=31) for 24 weeks. The primary endpoint is objective response rate (ORR) at week 25, as measured by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 by blinded independent central review in the intent-to-treat (ITT) population. Secondary endpoints include tumor volume score, active range of motion, stiffness by Numeric Rating Scale (NRS), pain by Brief Pain Inventory (BPI), and physical function measured by Patient-Reported Outcomes Measurement Information System (PROMIS). After the double-blind Part 1, eligible patients could continue to the open-label Part 2 for up to 24 weeks of dosing, results of which are expected in mid-2025. Patients who complete Part 2 may then enter the open-label extension phase (Part 3) for extended treatment and safety follow-up. About Pimicotinib (ABSK021)Pimicotinib (ABSK021), which is being developed by Abbisko Therapeutics, is a novel, orally administered, highly selective and potent small-molecule inhibitor of CSF-1R. Pimicotinib was recently granted Priority Review by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) for the treatment of patients with tenosynovial giant cell tumor (TGCT) who require systemic therapy. Pimicotinib has been granted breakthrough therapy designation (BTD) by China National Medical Products Administration (NMPA) and the US Food and Drug Administration (FDA), and priority medicine (PRIME) designation from the European Medicines Agency (EMA). Merck KGaA, Darmstadt, Germany, holds worldwide commercialization rights for pimicotinib. Advancing the Future of Cancer CareAt Merck KGaA, Darmstadt, Germany, we strive every day to improve the futures of people living with cancer. Building on our 350-year global heritage as pharma pioneers, we are focusing our most promising science to target cancer's deepest vulnerabilities, pursuing differentiated molecules to strike cancer at its core. By developing new therapies that can help advance cancer care, we are determined to create a world where more cancer patients will become cancer survivors. Learn more at About Merck KGaA, Darmstadt, GermanyMerck KGaA, Darmstadt, Germany, a leading science and technology company, operates across life science, healthcare and electronics. More than 62,000 employees work to make a positive difference to millions of people's lives every day by creating more joyful and sustainable ways to live. From providing products and services that accelerate drug development and manufacturing as well as discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices – the company is everywhere. In 2024, Merck KGaA, Darmstadt, Germany, generated sales of € 21.2 billion in 65 countries. The company holds the global rights to the name and trademark "Merck" internationally. The only exceptions are the United States and Canada, where the business sectors of Merck KGaA, Darmstadt, Germany, operate as MilliporeSigma in life science, EMD Serono in healthcare and EMD Electronics in electronics. Since its founding in 1668, scientific exploration and responsible entrepreneurship have been key to the company's technological and scientific advances. To this day, the founding family remains the majority owner of the publicly listed company. All Merck KGaA, Darmstadt, Germany, press releases are distributed by e-mail at the same time they become available on the EMD Group website. In case you are a resident of the USA or Canada, please go to to register for your online, change your selection or discontinue this service. View source version on Contacts Media Relations Phone: +1 (781) 427-4351 Investor Relations Phone: +49 6151 72-3321 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
Cision Canada
02-05-2025
- Health
- Cision Canada
Health Canada accepts semaglutide 2.4 mg, a GLP-1RA treatment for MASH, as a supplemental New Drug Submission under the Priority Review Policy
It is estimated that by 2030 the prevalence of MASH (Metabolic Dysfunction-Associated Steatohepatitis) in the Canadian general population will increase from 5.2% to 6.5%. 1 Left untreated, MASH can progress to serious and even fatal outcomes, such as cirrhosis, liver cancer, and the need for liver transplant. 2 MISSISSAUGA, ON, May 2, 2025 /CNW/ - Today, Novo Nordisk Canada announced that Health Canada has accepted for review, under the Priority Review policy, its supplemental new drug submission (sNDS) for semaglutide 2.4 mg, a once weekly therapy for people living with MASH (metabolic dysfunction-associated steatohepatitis). "Semaglutide 2.4 mg represents a therapeutic advancement for patients living with MASH, addressing a critical unmet medical need," says Vince Lamanna, President of Novo Nordisk Canada Inc. "Health Canada's acceptance of the Semaglutide 2.4 mg submission for review brings us one step closer to providing the first treatment option for eligible people living with MASH." About semaglutide 2.4 mg Semaglutide injection 2.4 mg is currently approved by Health Canada for chronic weight management and non-fatal myocardial infarction (MI) risk reduction in adults with established cardiovascular disease and BMI ≥ 27 kg/m 2. 3 Semaglutide is not approved in Canada for the treatment of MASH. Safety and efficacy are not established, and there is no guarantee that semaglutide will become commercially available for this use under clinical investigation. About Metabolic Dysfunction-Associated Steatohepatitis (MASH) MASH is a chronic, progressive metabolic disease affecting the liver, which can be potentially life-threatening if not properly managed. 4, 5 Among people who are living with overweight or obesity, approximately one in three also have MASH. 6 Excess fat can build up in the liver, which, over time, can lead to inflammation and severe scarring of the liver. 7 People living with MASH often experience few or no specific symptoms in the early stages of the disease, which often results in a delayed diagnosis. 8 It is estimated that by 2030 the prevalence of MASH in the Canadian general population will increase from 5.2% to 6.5%. 1 In Canada, the number of deaths are expected to double in people living with MASH by 2030. 1 About Novo Nordisk Novo Nordisk is a leading global healthcare company founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat serious chronic diseases built upon our heritage in diabetes. We do so by pioneering scientific breakthroughs, expanding access to our medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 76,300 people in 80 countries and markets its products in around 170 countries. For more information, visit Facebook, Instagram, X, LinkedIn and YouTube. SOURCE Novo Nordisk Canada Inc.
