Clinical Trial Updates
UK MHRA approves MSD’s pulmonary arterial hypertension therapy
MSD has received the UK Medicines and Healthcare products Regulatory Agency (MHRA) approval for sotatercept, marketed as Winrevair, to treat pulmonary arterial hypertension (PAH) in the adult population. The therapy is used along with other PAH medications to improve exercise capacity in patients with moderate to severe limitations in physical activity. Read More
Ractigen Therapeutics Announces First Patient Dosed in Phase I Clinical Trial for RAG-17 in SOD1-ALS
Ractigen Therapeutics, a clinical-stage pharmaceutical company dedicated to developing RNA-based innovative therapies, announced the successful dosing of the first patient in the Phase I clinical trial of RAG-17, an innovative siRNA therapy targeting Amyotrophic Lateral Sclerosis (ALS) associated with superoxide dismutase 1 (SOD1) gene mutations at Second Affiliated Hospital of Zhejiang University School of Medicine. Read more.
Novocure Announces Positive Topline Results from Phase 3 PANOVA-3 Clinical Trial of Tumor Treating Fields (TTFields) Therapy for Pancreatic Cancer
Novocure announced today that the pivotal, Phase 3 PANOVA-3 trial met its primary endpoint, demonstrating a statistically significant improvement in median overall survival (mOS) versus control. PANOVA-3 evaluated the use of Tumor Treating Fields (TTFields) therapy concomitantly with gemcitabine and nab-paclitaxel as a first-line treatment for unresectable, locally advanced pancreatic adenocarcinoma. Read more.
Nervonik Completes First-in-Human Clinical Trial of Nerve Stimulation Technology for Chronic Pain Treatment
Nervonik, a medical device startup company focusing on peripheral nerve stimulation (PNS) for the treatment of chronic pain, has successfully completed the first-in-human clinical study. PNS is a rapidly growing therapy space for the multibillion-dollar chronic pain market. The Nervonik PNS device utilizes proprietary technology, originally developed in Dr. Aydin Babakhani’s laboratory at the University of California, Los Angeles, to enhance therapy outcomes. Read more.
Resalis Therapeutics Announces Initiation of Phase 1 Clinical Trial for RES-010 in Obesity
Resalis Therapeutics announced the initiation of its firstin-human, Phase 1 study for RES-010, a non-coding RNA-based compound designed to provide a disease-modifying approach to obesity treatment. Preclinical studies have demonstrated that RES-010 reduces fat mass, preserves lean body mass, and enhances energy expenditure. By targeting fat reduction across various regions of the body, including visceral and hepatic stores, RES-010 has the potential to complement existing therapies such as GLP-1 receptor agonists, and support sustainable, long-term weight management. The Phase 1 trial (EUCT No: 2024- 514871-17-00) will explore the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of RES-010 in healthy, overweight, and moderately obese volunteers. Read More.
NIH Study Finds Tecovirimat Was Safe but Did Not Improve Mpox Resolution or Pain
The antiviral drug tecovirimat did not reduce the time to lesion resolution or have an effect on pain among adults with mild to moderate clade II mpox and a low risk of developing severe disease, according to an interim data analysis from the international clinical trial called the Study of Tecovirimat for Mpox (STOMP). There were no safety concerns associated with tecovirimat. Read More.
NMD Pharma publishes new data on the role of ClC-1 inhibition in Charcot-Marie-Tooth in the Annals of Clinical and Translational Neurology
Results from the ESTABLISH1 observational study confirms that neuromuscular junction (NMJ) dysfunction is an underappreciated disease characteristic in patients with Charcot-Marie-Tooth disease (CMT) types 1 and 2 affecting muscle function in patients. Additionally, results from preclinical studies provide proof-of-mechanism for recovery of muscle function with ClC-1 inhibition using NMD670 in CMT mouse models. Read More.
Soligenix Initiates Confirmatory Phase 3 Clinical Trial of HyBryte™ for the Treatment of Cutaneous T-Cell Lymphoma
Soligenix, Inc., a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that it has opened patient enrollment for its confirmatory Phase 3 study evaluating HyBryte™ (synthetic hypericin) in the treatment of cutaneous T-cell lymphoma (CTCL). The confirmatory Phase 3 study (Fluorescent Light Activated Synthetic Hypericin 2, FLASH2), builds on the previous statistically significant Phase 3 (FLASH) study, as well as a recent successful comparative study (HPN-CTCL-04) and an ongoing investigator-initiated study, each further supporting the design of the FLASH2 clinical trial. Read More.
Mayo Clinic’s largest-ever exome study offers blueprint for biomedical breakthroughs
Mayo Clinic’s Center for Individualized Medicine has achieved a significant milestone with its Tapestry study, generating the clinic’s largest-ever collection of exome data, which include genes that code for proteins — key to understanding health and disease. Read More.
Novartis intrathecal onasemnogene abeparvovec Phase III study meets primary endpoint in children and young adults with SMA
Novartis announced positive topline results from the Phase III STEER study. This pivotal study assessed the efficacy and safety of investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment-naïve patients with spinal muscular atrophy (SMA) Type 2, aged two to less than 18 years who are able to sit but have never walked independently. Efficacy and safety results for OAV101 IT were compared against a sham control, a procedure designed to mimic the administration of an investigational drug, without delivering any active treatment. Read More.
- The Phase III STEER study met its primary endpoint showing an increase from baseline in HFMSE total score in patients with SMA treated with intrathecal onasemnogene abeparvovec (OAV101 IT)
- OAV101 IT is the first investigational gene therapy to provide clinical benefit in treatment-naïve patients with SMA aged two and older with a positive risk benefit profile
- Novartis plans to share results with regulatory agencies in 2025 with the aim to make OAV101 IT available to help patients with SMA in need.
Novel Mini-Protein Plays a Role in Targeting Cancer Cells
By delivering a therapeutic dose of radiation via a novel mini-protein called AKY-1189, a team led by researchers in South Africa was able to selectively target cancer cells expressing the protein Nectin-4, found on the surface of most solid tumors, while avoiding nearby healthy tissue. This new radiopharmaceutical approach demonstrated “remarkable antitumor efficacy” with substantial radiation uptake in urothelial carcinoma and other Nectin-4 expressing tumors found in breast, lung, colorectal, and cervical cancers. No significant treatment-related adverse side effects were observed. Read More.
Sensorion completes first cohort enrolment in Phase I/II gene therapy trial
Sensorion has completed subject enrolment in the first cohort of Phase I/II Audiogene trial of gene therapy injection, SENS-501 for treating Otoferlin (OTOF) gene-mediated hearing loss. The trial is designed to assess the tolerability, efficacy, and safety of the gene therapy to address hearing loss in a specific group of infants and toddlers aged six to 31 months who have not received cochlear implants. Read More.
Reviewing Major Dermatologic Studies of the Year: 2024
As dermatology continues to evolve, 2024 brought groundbreaking studies that have reshaped clinical practices and deepened an understanding of skin health. From innovative therapies to insights into complex skin conditions, these studies have set new benchmarks for patient care and research. In this recap, explore the most impactful dermatologic studies of the year. Read More.
- Ligelizumab showed significant improvement in chronic spontaneous urticaria symptoms, though it did not surpass omalizumab in efficacy.
- Elevated lipid-related metabolites are causally linked to increased androgenic alopecia risk, suggesting lipid profile management in treatment.
- Hidradenitis suppurativa is associated with autoimmune and autoinflammatory diseases, revealing novel comorbidities and insights into its pathophysiology.
- AI in dermatology holds potential for enhanced diagnostic accuracy, though integration into clinical practice remains challenging.
- Oral dutasteride is identified as a first-line treatment for frontal fibrosing alopecia, demonstrating high efficacy and a favorable safety profile.
2024 Highlights: Progress in Ophthalmology Research
The year 2024 witnessed significant advancements in ophthalmology, with researchers and clinicians making strides in targeted and innovative therapies. Key developments included breakthroughs in cellular and genetic treatments, highlighting their potential to address unmet needs in eye care. Read this article by CGTLive which highlights some of the most notable updates in ophthalmology from 2024, offering insights into the trends and progress that defined the year. Readers can explore detailed coverage of these developments through the links provided. Read More.
Regulatory
CDSCO brings clinical trial site addition and change of PI applications to online mode
The Central Drugs Standard Control Organisation (CDSCO) has sought the industry to start submitting the applications for clinical trial site addition and change of principal investigator through online mode, as part of streamlining the regulatory submission procedure. The applicants should submit their applications for these purposes through the Sugam portal along with the checklist of documents and the approval of the ethics committee. Read More
FDA Refines Accelerated Approval Pathway with New Draft Guidance
The US Food and Drug Administration (FDA) has released a new draft guidance titled “Expedited Program for Serious Conditions — Accelerated Approval of Drugs and Biologics Guidance for Industry” refining the accelerated approval pathway for drugs and biologics targeting serious or life-threatening conditions. This guidance focuses on improved accountability, earlier confirmatory studies and greater clarity on novel endpoints.
Note: This newsletter includes links to published news articles and press releases, which may retain the original titles and summaries. The views and opinions expressed in these articles are those of the original author(s) and do not necessarily reflect the perspective of Lambda Therapeutic Research Ltd.
Lambda Therapeutic Research
Lambda Therapeutic Research stands at the forefront of the global Clinical Research Organization (CRO) landscape, headquartered in Ahmedabad, India, and extending its impact across Mehsana (India), Warsaw (Poland), London (UK), Barcelona (Spain), Toronto (Canada), Pittsburgh (USA), and Las Vegas (USA).
Our unwavering mission revolves around providing comprehensive end-to-end clinical research services to the global innovator, biotech, and generic pharmaceutical industries. Lambda has consistently raised the bar in the Clinical Research sector, achieving milestones through both organic growth and strategic acquisitions. In 2019, we strategically bolstered our North American presence by acquiring Novum Pharmaceutical Research Services, a distinguished 50-year-old CRO. Operating under the unified brand of Novum Pharmaceutical Research Services in North America, Lambda reinforces its position as a key player in the global CRO market.
Prioritizing secure IT infrastructure and automation, Lambda ensures timely project delivery, adhering to global regulatory standards. Our outstanding regulatory track record is underscored by over 60 successful international inspections and audits conducted by esteemed authorities, including the USFDA, EMEA, MHRA, EU member states, and other global bodies over the past five years.
Lambda’s dedication to excellence has garnered widespread recognition, with accolades such as the distinction of ‘Best Indian CRO’ by Frost & Sullivan (USA) and the prestigious title of ‘Great Indian Workplace’ by UBS Transformance. In the latest achievements, Lambda Therapeutic Research proudly received the ‘Regulatory Excellence’ Award at the CPhI Awards 2023 and the ‘Industry Partner of the Year’ Award at the Global Generics & Biosimilar Awards 2023. These honors reflect our ongoing commitment to regulatory compliance, industry leadership, and impactful collaborations within the pharmaceutical and clinical research sectors.
