PharmaFocus Today – Page 214 – media

Vanda expands commercial presence with $100M deal for US rights to J&J multiple sclerosis drug

Vanda Pharmaceuticals has a new commercial asset under its belt after striking a deal to buy certain rights to Johnson & Johnson’s multiple sclerosis med Ponvory for $100 million. Vanda secured U.S. and Canadian rights to the daily oral selective sphingosine-1-phosphate receptor 1 (S1P1R) modulator from J&J’s Actelion subsidiary. Ponvory was approved in 2021 to treat relapsing forms of multiple sclerosis and could be a potential treatment for a group of inflammatory and autoimmune disorders, including psoriasis and ulcerative colitis, Vanda said in its Thursday press release. The buy is a “significant milestone” for Vanda, CEO and chairman Mihael H. Polymeropoulos, M.D., said in a statement. The deal “expands our commercial portfolio and gives us access to a versatile immune response modifier that can potentially have broad application in treating a number of autoimmune-based disorders,” the chief added. J&J’s sale of certain Ponvory rights comes after Fierce Pharma in February ...
- Source: drugdu
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- December 11, 2023
FDA fast tracks Solid Biosciences’s Duchenne gene therapy

The FDA has granted fast track designation to Solid Biosciences’s gene therapy SGT-003 for the treatment of DMD. The US-based company received clearance from the FDA to start Phase I/II trial for the gene therapy on 14 November. SGT-003 uses a novel capsid AAV-SLB101 to deliver the DNA sequence encoding the shortened form of the dystrophin protein (microdystrophin). It also has R16 and R17 nNOS binding protein domains. DMD is a rare genetic condition that causes progressive muscle weakness. It affects approximately six in 100,000 individuals across Europe and North America, as per the US Muscular Dystrophy Association. The first gene therapy for treating DMD was approved earlier this year. In June, Sarepta Therapeutics’s gene therapy Elevidys (delandistrogene moxeparvovec-rokl) received accelerated approval by the FDA for treating ambulatory patients aged 4-5 years with a confirmed mutation in the DMD gene. There has been an increased interest in developing gene therapies ...
- Source: drugdu
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- December 11, 2023
Roche shares positive late-stage results for inavolisib combination in advanced breast cancer

Roche has shared positive results from a late-stage study of its investigational therapy, inavolisib, as part of a combination treatment for advanced breast cancer. The phase 3 INAVO120 trial has been evaluating the targeted drug alongside Pfizer’s CDK4/6 inhibitor Ibrance (palbociclib) and fulvestrant as a potential first-line treatment option for patients with PIK3CA-mutated, hormone receptor (HR)-positive, HER2-negative, endocrine-resistant, locally advanced or metastatic breast cancer. The study met its primary endpoint, demonstrating a statistically significant and clinically meaningful improvement in progression-free survival compared to palbociclib and fulvestrant alone. A “clear positive trend” towards improved survival was also observed for the inavolisib combination, Roche said, although overall survival data was immature at the time of the analysis and will continue to be analysed. Breast cancer is the most common cancer worldwide, with more than 290,000 people in the US expected to be diagnosed with the disease in 2023. HR-positive breast cancer is ...
- Source: drugdu
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- December 11, 2023
Study identifies new amyloid-forming protein linked to early-onset dementia

A new study led by the Medical Research Council’s (MRC) Laboratory of Molecular Biology (LMB) has identified a new protein as a potential therapeutic target for frontotemporal dementia, a type of early-onset dementia. Dementia is a progressive neurodegenerative disorder that affects more than 944,000 people in the UK. Frontotemporal dementia occurs from the degeneration of the frontal and temporal lobes of the brain, which control emotions, personality and behaviour, along with speech. Most neurodegenerative diseases involve proteins aggregating into filaments known as amyloids. When identified, researchers can target these proteins for diagnostic tests and treatments. However, in around 10% of frontotemporal dementia cases, researchers had to identify the TAF15 protein. Published in Nature, MRC LMB researchers successfully pinpointed the aggregated structures of the protein using cutting-edge cryo-electron microscopy to study the brains of four patients who had this type of early-onset dementia. The FUS protein is already known to be ...
- Source: drugdu
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- December 11, 2023
Laboratory trade group, providers oppose FDA’s lab developed test proposal

Dive Brief The Food and Drug Administration received more than 19,600 comments related to its planned changes to the oversight of laboratory developed tests (LDTs). The public comment period closed on Monday. Multiple groups pushed back against plans to increase FDA oversight of LDTs. The American Clinical Laboratory Association (ACLA) is at the forefront of the resistance, submitting a 107-page document that outlines “grave concerns” and urges the FDA to withdraw the proposal. Hospitals and healthcare provider groups, like the American Hospital Association (AHA), also opposed the FDA’s proposal, arguing that the rule would limit patients’ access to tests. Dive Insight The FDA closed the comment period Monday, resisting calls to give affected groups more time to send feedback. The length of the comment period is one of many points of contention. Plans to increase regulatory oversight of LDTs, without gaining additional powers from Congress, have alarmed a spectrum of ...
- Source: drugdu
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- December 9, 2023
Large language models enhance differential diagnosis, paving the way for AI-assisted medical decision-making

A recent study published in the ArXiv preprint* server discusses the optimization of large language models (LLMs) for accurate differential diagnosis (DDx). Background Accurate diagnosis is the first step in effective medical care. It has been perceived that artificial intelligence (AI)-based models can be used to assist clinicians for accurate diagnosis of a disease. The real-world diagnostic process involves an interactive and iterative process with rational reasoning about a DDx. A physician weighs different diagnostic possibilities based on varied clinical information procured from advanced diagnostic procedures. Deep learning has been applied to the generation of DDx in ophthalmology, dermatology, and radiology. Due to the absence of interactive capabilities, deep learning models cannot assist patients with diagnosis through fluent communication in their native language. This interactive shortcoming can be overcome with the development of LLMs, which can be used to design effective tools for DDx. LLMs are trained using a massive ...
- Source: drugdu
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- December 9, 2023
Microbot Medical is a step closer to an FDA IDE submission

BY CHRIS NEWMARKER Microbot Medical today announced the successful completion of its pivotal pre-clinical study of its Liberty endovascular robotic surgical system. The study, performed under good laboratory practice (GLP) and essential for the company’s investigational device exemption (IDE) submission, took place under rigorous FDA guidelines. The study involved pigs. In this study, three interventional radiologists performed 96 robotic navigations using Liberty. They used the system to control various commercially available intravascular catheterization devices. They then microscopically examined and evaluated the target vessels and surrounding tissue. Microbot Medical officials think Liberty’s remote operation could enable it to democratize endovascular interventional procedures. “I am very pleased with the positive outcomes of the histopathology report and the completion of the GLP study,” said Juan Diaz Cartelle, chief medical officer of Braintree, Massachusetts–based Microbot Medical. “This gives us confidence to move forward to the next stage of human clinical study.” Microbot Medical CEO ...
- Source: drugdu
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- December 9, 2023
ChatGPT Does a Bad Job of Answering People’s Medication Questions

Study Finds Researchers recently tested ChatGPT’s ability to answer patient questions about medication, finding that the AI model gave wrong or incomplete answers about 75% of the time. Providers should be wary of the fact that the model does not always give sound medical advice, given many of their patients could be turning to ChatGPT to answer health-related questions. By KATIE ADAMS Researchers recently tested ChatGPT’s ability to answer patient questions about medication, finding that the viral chatbot came up dangerously short. The research was presented at the American Society of Health-System Pharmacists’ annual meeting, which was held this week in Anaheim. The free version of ChatGPT, which was the one tested in the study, has more than 100 million users. Providers should be wary of the fact that the generative AI model does not always give sound medical advice, given many of their patients could be turning to ChatGPT ...
- Source: drugdu
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- December 9, 2023
Sanofi’s quiet Sarclisa chalks up a first-in-class win with eyes on a first-line multiple myeloma nod

In the currently mismatched CD38 antibody race, Sanofi’s Sarclisa has delivered a pivotal trial win, which might help the lagging follower level the playing field a little. Using Sarclisa on top of Takeda’s Velcade, Bristol Myers Squibb’s Revlimid and the steroid dexamethasone (VRd), significantly reduced the risk of disease progression or death versus VRd alone in patients with newly diagnosed multiple myeloma not eligible for transplant, Sanofi said Thursday. Sanofi’s phase 3 IMROZ readout comes as Sarclisa’s in-class rival, Johnson & Johnson’s Darzalex, is sharing a positive phase 3 for its VRd combo in transplant-eligible patients at the upcoming American Society of Hematology annual meeting. Still, the J&J data don’t prevent Sanofi from claiming the first phase 3 win for an anti-CD38 antibody with VRd in transplant-ineligible patients. Results from the IMROZ trial will be shared at a future medical meeting and will support a regulatory filing, Sanofi said. A ...
- Source: drugdu
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- December 9, 2023
Pfizer and Arvinas plan vepdegestrant programme expansion after data at SABCS.

Pfizer and Arvinas are broadening their vepdegestrant programme to include more settings, as positive data is reported from the Phase Ib study of the drug to treat breast cancer. Vepdegestrant is an orally administered protein degrader designed as a proteolysis targeting chimera (PROTAC) that targets both wild-type and mutant oestrogen receptor (ER) found in some breast cancers. The collaboration between the two companies began in July 2021 in a $2.4bn deal to develop the PROTAC drug. Under the terms of the agreement, both firms would co-develop and market vepdegestrant. In the abstract on data from a Phase Ib study (NCT04072952) presented at the ongoing 2023 San Antonio Breast Cancer Symposium (SABCS), the reported clinical benefit rate (CBR) was 37.1% with 200mg and 38.9% with 500mg of the drug amongst 71 patients who received either dose (abstract no. Abstract GS3-03). The treatment was generally well tolerated, with the most common side ...
- Source: drugdu
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- December 9, 2023

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