Ipsen Partners with Skyhawk Therapeutics for RNA-Focused Research in Rare Neurological Disorders

Ipsen and Skyhawk Therapeutics have entered into an exclusive global partnership to explore and create new small molecules that can influence RNA for rare neurological conditions. Under this agreement, Ipsen has the option to obtain exclusive rights worldwide for developing promising candidates resulting from the collaboration. Once successful candidates are identified, Ipsen will oversee all subsequent activities. Skyhawk’s innovative platform expedites the development of small molecules targeting RNA in various therapeutic domains, particularly focusing on rare neurological disorders.

“We’re thrilled to collaborate with the skilled professionals at Skyhawk as we investigate the possibility of altering RNA expression in rare and severe neurological disorders,” stated Steve Glyman, Senior Vice President and Head of Neuroscience, Research & Development at Ipsen. “Our dedication and proficiency in addressing movement disorders, along with our extensive portfolio, aim to provide innovative and leading treatments to individuals facing significant unmet medical needs. This partnership leverages a groundbreaking platform at the forefront of scientific research, enhancing our efforts to make meaningful advancements.”

Sergey Paushkin, the Chief Scientific Officer at Skyhawk, expressed enthusiasm about the collaboration with Ipsen, emphasizing their mutual dedication to advancing patient care. Paushkin highlighted their joint commitment to enhancing the range of groundbreaking therapies in Ipsen’s pipeline, particularly focusing on addressing rare neurological conditions lacking approved treatments.”

According to the agreement, Skyhawk stands to gain a maximum of $1.8 billion through various stages such as development, regulatory processes, and commercial achievements. This includes an initial payment for the option and research collaboration, along with the possibility of earning graduated royalties.

SynOx Therapeutics Secures $75 Million to Advance Best-in-class TGCT Treatment to Phase III Trial

SynOx Therapeutics Limited has just wrapped up a $75 million Series B financing round, which saw participation from Forbion, HealthCap, and new investor Bioqube Ventures. The funding will be directed towards generating pivotal Phase III clinical and CMC (Chemistry, Manufacturing, and Controls) data for emactuzumab, SynOx’s promising monoclonal antibody designed to inhibit CSF-1(R) and potentially become the leading treatment for Tenosynovial Giant Cell Tumour (TGCT). TGCT, a form of tumor affecting the soft tissue surrounding joints and tendons, is a severely debilitating condition, particularly impacting major joints like the knee, hip, and ankle.

Ray Barlow, CEO of SynOx Therapeutics, expressed that this marks a pivotal moment for the company. The significant funding received will enable them to gather crucial data for emactuzumab in treating TGCT. He emphasized the unique qualities of emactuzumab, highlighting its efficacy, swift onset, and sustained response, which distinguishes it from other treatments under development. Barlow believes that emactuzumab will offer a valuable solution for patients grappling with this severe illness.

Emactuzumab represents an innovative advancement as a CSF-1R mAb, boasting a potentially superior profile compared to existing options. Previous clinical investigations focusing on TGCT have highlighted its noteworthy performance, showcasing a remarkable objective response rate of 71%, along with swift and substantial tumor regression, sustained efficacy, and notable enhancements in patients’ functional capabilities. Crucially, these studies underscored emactuzumab’s favorable tolerability and manageable safety profile. SynOx is now embarking on a Phase III trial (TANGENT) to evaluate the efficacy and safety of emactuzumab in individuals with both localized and diffuse TGCT.

Roche’s Alecensa Receives FDA Nod as First Adjuvant Treatment for ALK-positive Early-stage Lung Cancer

Roche has declared that the FDA approved Alecensa® (alectinib) to be used as adjuvant therapy after tumor removal in patients diagnosed with anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC) (tumors measuring ≥ 4 cm or those with positive lymph nodes), as detected by an FDA-endorsed test. Alecensa marks the inaugural and singular ALK inhibitor sanctioned for individuals with ALK-positive early-stage NSCLC who have undergone tumor resection surgery.

“The approval of Alecensa represents a significant milestone for individuals recently diagnosed with early-stage ALK-positive lung cancer. Previously, they lacked access to ALK-specific therapy,” noted Ken Culver, Director of Research and Clinical Affairs at ALK Positive, Inc. “These patients, typically diagnosed at a younger age, often confront recurrence and a heightened risk of brain metastases compared to other NSCLC types. With this major breakthrough, it’s crucial that all early-stage lung cancer patients undergo ALK and other recommended biomarker testing to ensure they receive the most suitable treatment.”

The approval is rooted in favorable outcomes from the Phase III ALINA study, indicating that Alecensa significantly decreased the likelihood of disease recurrence or death by 76% compared to platinum-based chemotherapy in individuals with completely resected IB to IIIA ALK-positive NSCLC (tumor ≥ 4 cm). Additionally, there was an observed enhancement in central nervous system disease-free survival. The safety profile of Alecensa in this study remained consistent with prior trials in metastatic settings, with no unexpected safety concerns. These findings were presented at the European Society of Medical Oncology Congress 2023 Presidential Symposium and subsequently published in the New England Journal of Medicine in April 2024.

Phase III SELECT-GCA Trial Highlights Upadacitinib (RINVOQ) Efficacy in Giant Cell Arteritis Management

AbbVie reported positive initial findings from SELECT-GCA, a Phase III trial conducted across multiple centers, where upadacitinib (marketed as RINVOQ®; taken at a dose of 15 mg once daily) when used alongside a 26-week gradual reduction of steroids, met its primary goal of maintaining remission from week 12 to week 52 in adults diagnosed with giant cell arteritis (GCA). The study revealed that 46 percent of participants treated with upadacitinib 15 mg alongside the steroid taper regimen achieved sustained remission, compared to 29 percent of those who received a placebo alongside a 52-week steroid taper regimen. This difference was statistically significant with a p-value of 0.0019.

Kori Wallace, M.D., Ph.D., vice president and global head of immunology clinical development at AbbVie, stated that numerous individuals affected by GCA are enduring the severe symptoms of the illness, despite the few treatment choices accessible to them. She emphasized AbbVie’s dedication to enhancing the quality of life for those with immune-related conditions by creating novel therapies to address significant gaps in medical care.

Giant-cell arteritis falls under the category of large-vessel vasculitis, although it also affects medium and small arteries, notably the superficial temporal artery, which is why it’s often called temporal arteritis. As per DelveInsight’s analysis, it was observed that across the 7MM, patients in the age segment >80 years accounted for the highest number of prevalent cases of giant-cell arteritis in 2023.

The study successfully met key secondary goals, with notable outcomes. More patients who received upadacitinib 15 mg alongside a 26-week steroid taper regimen achieved sustained complete remission from week 12 through week 52 compared to those who received a placebo alongside a 52-week steroid taper regimen (37% versus 16%; p<0.0001). Additionally, fewer patients in the upadacitinib 15 mg group experienced disease flare-ups through week 52 compared to the placebo group (34% versus 56%; p=0.0014). However, it’s important to note that upadacitinib 7.5 mg did not meet either the primary or any of the secondary endpoints in the study.

AskBio’s AB-1002 Gene Therapy Program Receives FDA Fast-Track Designation for Congestive Heart Failure Treatment

Bayer AG and Asklepios BioPharmaceutical, Inc. revealed that the FDA has awarded Fast Track Designation for the AB-1002 program. AB-1002 is an experimental gene therapy administered to the heart aimed at stimulating the production of a continuously active version of protein inhibitor 1 (I-1c), which is intended to counteract the activity of protein phosphatase 1. By obstructing the function of this protein associated with congestive heart failure (CHF), there’s a potential for a beneficial impact on heart health.

“The FDA’s Fast Track Designation granted to AB-1002 marks a significant milestone in advancing this program’s clinical development, emphasizing our dedication to delivering potentially beneficial therapies to individuals suffering from advanced congestive heart failure,” stated Canwen Jiang, MD, PhD, who serves as both Chief Development Officer and Chief Medical Officer at AskBio. “We are eager to conclude our Phase II GenePHIT clinical trial, actively enrolling patients with severe heart failure, and remain steadfast in our pursuit of realizing the complete therapeutic potential of AB-1002 for addressing this debilitating condition.”

AB-1002 is a gene therapy under investigation that hasn’t been approved for marketing yet, and its effectiveness and safety haven’t been confirmed or thoroughly assessed. AskBio is presently recruiting participants for the Phase II GenePHIT trial, testing AB-1002 (also referred to as NAN-101) for treating CHF.