Globenewswire | July 20, 2023
Catalyst Pharmaceuticals, Inc. announced the completion of its acquisition from Santhera Pharmaceuticals Holdings of an exclusive license for North America for vamorolone, a potential treatment for patients suffering with Duchenne Muscular Dystrophy. The license is for exclusive commercial rights in the U.S., Canada, and Mexico, as well as the right of first negotiation in Europe and Japan should Santhera pursue partnership opportunities. Additionally, Catalyst will hold North American rights for any future approved indications of vamorolone.
Vamorolone is a promising best-in-class dissociative anti-inflammatory steroid treatment for Duchenne Muscular Dystrophy ("DMD"). In clinical studies, vamorolone demonstrated efficacy with a significant reduction of steroid-associated side effects and benefits for bone health, growth, and behavior, offering the potential to address an important unmet medical need in DMD patients. Vamorolone has received FDA Orphan Drug and Fast Track designations and has been granted a PDUFA action date of October 26, 2023.
"With the addition of vamorolone, we have accomplished another important milestone in the execution of our portfolio expansion strategy," stated Patrick J. McEnany, Chairman and CEO of Catalyst. "The license for vamorolone reflects our strategic focus on opportunities where we can not only apply novel technology to address critical unmet patient needs, but where we can leverage Catalyst's existing integrated capabilities and infrastructure to commercialize the drug efficiently. We believe that vamorolone, if approved, has the potential to deliver significant near and long-term value and to be a very meaningful advancement to the current standard of care paradigm for DMD patients."
Mr. McEnany continued, "Vamorolone fortifies our neuromuscular portfolio with an innovative therapy that, in clinical studies, demonstrated an enhanced safety and tolerability profile as compared to prednisone. We plan to include vamorolone for DMD patients in our Catalyst Pathways® specialty pharmacy program to help ensure that all patients have access to the full patient benefits of the program. Our planned strategy to facilitate access to vamorolone underscores our steadfast commitment to improving the lives of patients suffering from rare neurological conditions."
Duchenne Muscular Dystrophy, or DMD, the most common form of muscular dystrophy, is a rare, fatal neuromuscular disorder characterized by progressive muscular dysfunction, leading to loss of ambulation, respiratory failure, and death. Corticosteroids are the current standard of care for treating DMD; however, this treatment is associated with significant side effect burdens. The U.S. prevalence for DMD is estimated to be between 11,000 and 13,000 patients. Of patients currently being treated for DMD, approximately 75% receive concomitant steroid treatment.
Vamorolone Commercial Operational Plan
Catalyst expects to launch vamorolone early in the first quarter of 2024, assuming regulatory approval on the PDUFA date of October 26, 2023. Catalyst anticipates minimal sales and marketing personnel expansion with fewer than 10 additional team members required, resulting from the exceptional synergy within its existing neuromuscular franchise. Catalyst plans to incorporate vamorolone for DMD into its Catalyst Pathways® specialty pharmacy program to ensure that patients have access to full patient benefits.
Vamorolone is an investigational drug candidate with a mode of action based on binding to the same receptor as glucocorticoids but modifying its downstream activity and as such, is considered a dissociative anti-inflammatory steroid drug [2-5]. This mechanism has shown the potential to 'dissociate' efficacy from steroid safety concerns, and therefore vamorolone could emerge as an alternative to existing corticosteroids, the current standard of care in children, adolescent, and adult patients with DMD. In the pivotal VISION-DMD study, vamorolone met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile . The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, vomiting, and vitamin D deficiency. Adverse events were generally of mild to moderate severity.
Vamorolone has been granted Orphan Drug status for DMD in the U.S. and Europe and has received Fast Track and Rare Pediatric Disease designations by the U.S. FDA and Promising Innovative Medicine (PIM) status from the UK MHRA for DMD. Vamorolone is an investigational medicine and is currently not approved for use by any health authority.
About Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy ("DMD") is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. DMD is characterized by muscle inflammation and damage which are present at birth or shortly thereafter. Inflammation leads to fibrosis of muscle and is clinically manifested by progressive muscle degeneration and weakness. Major milestones in the disease are the loss of ambulation, the loss of self-feeding, the start of assisted ventilation, and the development of cardiomyopathy. DMD reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure. Corticosteroids are the current standard of care for the treatment of DMD.
About Catalyst Pharmaceuticals
With exceptional patient focus, Catalyst is committed to developing and commercializing innovative first-in-class medicines that address rare neurological and epileptic diseases. Catalyst's U.S. commercial product portfolio consists of FIRDAPSE® (amifampridine) Tablets 10 mg, approved for the treatment of Lambert-Eaton myasthenic syndrome ("LEMS") for adults and children ages six to seventeen. In January 2023, Catalyst acquired the U.S. commercial rights of FYCOMPA® (perampanel) CIII, a prescription medicine approved in people with epilepsy aged four and older alone or with other medicines to treat partial-onset seizures with or without secondarily generalized seizures, and with other medicines to treat primary generalized tonic-clonic seizures for people with epilepsy aged 12 and older. Further, Canada's national healthcare regulatory agency, Health Canada, has approved the use of FIRDAPSE for the treatment of adult patients in Canada with LEMS.
PRNewswire | August 17, 2023
Frontage Laboratories, Inc. and its wholly-owned subsidiary, Frontage Canada, Inc., completed the acquisition of Nucro-Technics Inc. and its affiliate Nucro-Technics Holdings, Inc.
Nucro-Technics is a pharmaceutical contract research organization that conducts analytical chemistry, microbiology, toxicology, bioanalytical, and stability sample storage and testing services. In addition, it provides consulting services, especially in quality control and assurance as well as in Natural Health Product Regulations.
Nucro-Technics, located in the Toronto, Canada metropolitan area, operates in a state-of-the-art 60,000 square foot facility and employs personnel having expertise in testing both large and small molecules designed to treat various medical conditions. For over 50 years, Nucro-Technics has been conducting studies for various clients including major pharmaceutical, biotech, and medical device companies, as well as smaller organizations including industry trade groups and academia. Nucro-Technics has successfully developed and validated numerous methods, as well as participated in providing comprehensive IND packages to clients for regulatory submissions. Nucro-Technics, an ISO 9001:2015 certified organization, conducts studies that are in compliance with GLP and GMP regulations.
"Today marks a momentous and an exciting occasion. It is a day to celebrate two great companies coming together. It is with great pleasure that I welcome the extraordinary Nucro-Technics team to the Frontage family," said Dr. Abdul Mutlib, CEO of Frontage. "Nucro-Technics has been recognized as a leader in the Canadian CRO market for over 50 years, and its unparalleled client services is a perfect match for the culture of Frontage Laboratories. This acquisition not only significantly strengthens our presence in Canada, but also will enable the combined organization to offer a more expansive suite of services to clients across the globe. We are looking forward to partnering with the Nucro team to deliver the world-class expertise that clients have come to expect from us."
According to Dr. Song Li, Founder and Chairman of Frontage: "Nucro has developed a remarkable platform with a truly impressive team of experts. The closing of this transaction marks the next step in our ongoing effort to build Frontage into one of the major players in the global CRO market. In doing so, we will continue our focus on delivering the highest levels of quality, technology and expertise to our clients."
"I'm delighted to announce that we will be partnering with Frontage Labs for the next phase of Nucro Technics' growth journey. Since founding the business in 1970, we've worked hard to become the North American boutique in pre-clinical and drug product release testing services. Partnering with Frontage Labs marks an exciting chapter in the evolution of our efforts to support and accelerate pharmaceutical research and development," said John Fanaras of Nucro Technics.
Frontage was advised by Borden Ladner Gervais LLP (BLG) while Nucro-Technics was advised by Dentons and PricewaterhouseCoopers Corporate Finance Inc.
Frontage Holdings Corp (1521.HK), together with its wholly owned subsidiary Frontage Laboratories, Inc., is a global Contract Research Organization (CRO) which provides integrated, science-driven, product development services from drug discovery to late phase clinical process to enable biopharmaceutical companies to achieve their development goals. Comprehensive services include drug metabolism and pharmacokinetics, analytical testing and formulation development, preclinical and clinical trial material manufacturing, bioanalysis, preclinical safety and toxicology assessment and early phase clinical studies. Frontage has enabled many biotechnology companies and leading pharmaceutical companies of varying sizes to advance a myriad of new molecules through development and to successfully file global regulatory submissions.
prnewswire | September 21, 2023
Biofourmis, a leading global technology-enabled care delivery company, announced today the release of its expanded Digital Clinical Trials solution within the Biofourmis platform for biopharma and other life sciences companies. The solution offers robust digital tools and resources for trial sponsors and investigators aimed at accelerating drug development and improving access and diversity while also reducing costs.
Biofourmis' Digital Clinical Trials solution includes a technology-enabled, scalable platform and services that support digital tools enablement, trial decentralization, safety monitoring, personalized care, easier participant recruitment and more equitable access to treatment options for patients everywhere. The solution helps sponsors and researchers accelerate clinical development with remote data collection and novel digital endpoints from Biofourmis' library of biomarkers across multiple therapeutic areas.
The expanded solution offers a more holistic workflow, new data management automations and a seamless integration of essential clinical trial documentation, participant scheduling and data collection elements. Because the artificial intelligence (AI)-powered platform is highly configurable, study start-up timelines can be reduced from months to days. The connected platform seamlessly integrates with 40+ devices, enabling study teams the freedom to select the right device for the right disease area.
Biopharma companies can also tap into Biofourmis' growing principal investigator (PI) network. The network includes experienced and esteemed PIs from leading academic medical centers as a valuable resource to manage digitally driven research and accelerate the pace of recruitment with access to patient databases. Health systems that already use the Biofourmis platform for technology-enabled care delivery at home can easily and quickly expand their clinical research initiatives by leveraging the connected platform to include the Digital Clinical Trials functionality.
"We have consistently earned high satisfaction scores from both researchers and trial participants for our solutions. Now, we have identified additional ways to accelerate access to digital clinical trials, lower costs, and improve the experience for all stakeholders," said Biofourmis COO Jaydev Thakkar. "These new technology and service elements create a more holistic, turnkey solution that achieves those aims while offering greater synergies from drug development to commercialization and creating equitable access to treatment options and clinical research for patients everywhere."
Market-leading enhancements to the Digital Clinical Trials solution include
Robust eConsent and ePROs Capabilities: Robust screening and informed consent tools are integrated into workflows to accelerate recruitment, decrease enrollment time, and help ensure that participants fully understand risks and benefits associated with the trial. The documentation content is easily configurable and compliant with regulatory requirements. The enhanced ePROs (electronic patient-reported outcomes) collection capabilities drive adherence through automated reminders of participants' reporting requirements while offering them the ability to easily share outcomes beyond traditional clinical measures. Reporting flexibility offers investigators increased visibility into participant safety, health and experience.
Dynamic Participant Scheduling: Upon enrollment, the Digital Clinical Trials solution helps research stay on course while encouraging participant adherence and engagement through a virtual visit and schedule of assessment module that streamlines all the "to-do" items for the participant and caregiver, intuitively listing all the required patient reporting and external assessment tasks. Reminders and notifications help participants stay adherent; but if a task is missed, the schedule can automatically adjust so the participant can remain enrolled, and the trial can move forward.
InHome Services: As with all solutions within Biofourmis Connect and Care, In-Home Services such as nurse visits, phlebotomy, infusion, imaging, and other ancillary services delivered within the home are available via the connected platform to support decentralized clinical trials—which further reduces burden on investigators and participants.
Unmatched Safety Monitoring: The solution leverages AI to monitor the efficacy and side effects of drugs. A specific example includes pre-screening for clinical events triggered by high potassium levels (hyperkalemia), which can reduce clinical trial costs by decreasing frequent manual checks by medical professionals while still protecting patient safety. In addition, cardiac safety monitoring capabilities such as auto-detection of QTc prolongation (an extended interval between the heart contracting and relaxing) can improve the safety of trials by detecting patients earlier who are at risk of developing critical heart arrhythmias due to drug side effects.
"Leveraging digital tools and automating and accelerating trial processes for both researchers and participants can increase the likelihood that endpoints will be reached sooner, which lowers costs and simplifies tasks for everyone involved," Thakkar said. "More importantly, shorter trials with more meaningful results mean that groundbreaking new therapies can be discovered and developed faster to improve outcomes for patients worldwide."
Biofourmis brings the right care to every person, no matter where they are. The company's AI-driven solution collects and analyzes patient data in real time and identifies shifts that require proactive interventions. This vital innovation provides people everywhere with connected access to hospital-level services, virtual provider networks for remote care, in-home services, and life-changing clinical trials—all without leaving their homes. Trusted by leading health systems, payers, biopharma companies and patients alike, Biofourmis' connected platform improves patient outcomes, prevents hospital readmissions, accelerates drug development, and closes critical gaps in care—ultimately making science smarter, healthcare simpler, and patients healthier.