Submission marks major milestone for novel excipient BLG

Original title: “Zerion partner files first EU application for Dispersome‑enabled product”

A Lutathera copycat has become the first “radioligand equivalent” cleared by regulators. Elsewhere, Novo dropped the “Nordisk” from its name and a novel RNAi drug showed early promise.

Original title: “FDA clears first generic radioligand drug; Sionna turns to layoffs”

The U.S. Food and Drug Administration today announced the final design of the Expedited Investigational New Drug (IND) Pilot.

Original title: “FDA Launches Expedited IND Pilot, Begins Accepting Applications”

Analysts believe Cullinan Therapeutics and Taiho Oncology can overcome the safety risks as zipalertinib “sets [a] new bar” for first-line treatment of a type of lung cancer. The candidate is already under review for the the second-line setting, with a decision expected by early next year.

Original title: “Despite 3 patient deaths, Cullinan-Taiho plan FDA filing for lung cancer pill on survival benefit”

Guidance supports MRD‑guided treatment approach

Original title: “NICE backs daratumumab quadruplet for transplant‑eligible myeloma”

DelveInsight s Helicobacter Pylori Infection Pipeline Insight 2026 report provides comprehensive insights about 5 companies and 5 pipeline drugs in the Helicobacter Pylori Infection pipeline landscape It covers the Helicobacter Pylori Infection Pipeline drug profiles including clinical and ... DelveInsight s Helicobacter Pylori Infection Pipeline Insight 2026 report provides comprehensive insights about 5 companies and 5 pipeline drugs in the Helicobacter Pylori Infection pipeline landscape It covers the Helicobacter Pylori Infection Pipeline drug profiles including clinical and nonclinical stage ... Press release - DelveInsight Business Research LLP - Helicobacter Pylori Infection Clinical Trial Pipeline Gains Momentum: 5+ Companies Lead the Charge in Pioneering New Treatments | DelveInsight - published on openPR.com TenNor is currently conducting a multi-center, randomized, double-blind, bismuth-containing quadruple therapy-controlled Phase III study to evaluate the efficacy and safety of Rifasutenizole in the primary treatment of patients with H. pylori infection. If you're tracking ongoing Helicobacter Pylori Infection Clinical trials, this press release is a must-read @ https://www.delveinsight.com/sample-request/helicobacter-pylori-infections-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr The Helicobacter Pylori Infection Pipeline report provides insights into:- • The report provides detailed insights about companies that are developing therapies for the treatment of Helicobacter Pylori Infection with aggregate therapies developed by each company for the same. It covers the Angelman Syndrome Pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Angelman Syndrome Pipeline Therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space. Curious about the latest updates ... DelveInsight's, "Charcot Marie Tooth Disease Pipeline Insight, 2026" report provides comprehensive insights about 13+ companies and 15+ pipeline drugs in Charcot Marie Tooth Disease pipeline landscape.

Original title: “Helicobacter Pylori Infection Clinical Trial Pipeline Gains Momentum: 5+ Companies Lead the Charge in Pioneering New Treatments | DelveInsight”

DelveInsight s Charcot Marie Tooth Disease Pipeline Insight 2026 report provides comprehensive insights about 13 companies and 15 pipeline drugs in Charcot Marie Tooth Disease pipeline landscape It covers the Charcot Marie Tooth pipeline drug profiles including clinical and nonclinical ... Press release - DelveInsight Business Research LLP - Charcot Marie Tooth Disease Clinical Trial Pipeline Gains Momentum: 13+ Companies Lead the Charge in Pioneering New Treatments | DelveInsight - published on openPR.com By regulating this channel, it increases muscle excitability and enhances neuromuscular signal transmission. The therapy is being developed to improve muscle function and alleviate fatigue in patients with Charcot-Marie-Tooth disease. It is currently undergoing Phase II clinical evaluation for the treatment of Charcot-Marie-Tooth disease. @ https://www.delveinsight.com/sample-request/charcot-marie-tooth-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr Key Takeaways from the Charcot Marie Tooth Pipeline Report • In September 2026 - Elpida Therapeutics updated its clinical study for ELP-02, a potential treatment for Charcot-Marie-Tooth Disease Type 4J (CMT4J). The first-in-human Phase 1/2 study is currently recruiting and is evaluating the safety, tolerability, and efficacy of a single intrathecal dose of ELP-02 in patients with CMT4J. • In July 2026, Novartis updated its first-in-human Phase 1 clinical trial of EDK060 for Charcot-Marie-Tooth Disease Type 1A (CMT1A). It covers the PI3K Inhibitor pipeline drug profiles, including PI3K Inhibitor clinical and nonclinical stage products. It also covers the PI3K Inhibitor therapeutics assessment by product type, stage, PI3K Inhibitor route of administration, and PI3K Inhibitor molecule type. It further highlights the inactive PI3K Inhibitor pipeline products in ... DelveInsight's "Glaucoma Treatment Market Insights, Epidemiology and Market Forecast 2034" report offers an in-depth understanding of Glaucoma, historical and forecasted epidemiology, as well as the market trends in the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan.

Original title: “Charcot Marie Tooth Disease Clinical Trial Pipeline Gains Momentum: 13+ Companies Lead the Charge in Pioneering New Treatments | DelveInsight”

Etcamah is the second oral “SERD” to recently fall short in first-line breast cancer, following a similar setback from Roche earlier this year.

Original title: “AstraZeneca’s newly approved pill fails frontline breast cancer study”

Positive topline results from a second straight study in generalized anxiety disorder shift the narrative for Definium Therapeutics’ DT120 to commercialization, Jefferies analysts said Monday, while Stifel wrote that the asset is moving towards an FDA submission.

Original title: “Definium moves toward FDA submission for LSD drug in anxiety after ‘another clear win’”

Projected by some analysts as a future blockbuster, Isembyld is the first “muscle-targeted” treatment for the disease and, in clinical testing, helped improve motor function in people on standard therapies.

Original title: “Scholar Rock SMA drug approved by FDA”

/PRNewswire/ -- New results from the global Phase I/II ARROS-1 trial showed clinically meaningful activity with zidesamtinib in patients with advanced or... 14, 2026 /PRNewswire/ -- New results from the global Phase I/II ARROS-1 trial showed clinically meaningful activity with zidesamtinib in patients with advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC) who had not previously received a tyrosine kinase inhibitor (TKI). Treatment-related adverse events led to dose reductions in 11% of patients and treatment discontinuation in 1%. "Zidesamtinib demonstrated clinically meaningful activity in TKI-naive patients with ROS1-positive NSCLC, with a safety profile consistent with previous reports and low rates of dose reduction and discontinuation. The findings support continued investigation of zidesamtinib in earlier lines of therapy," said Alexander Drilon, M.D., of Memorial Sloan Kettering Cancer Center and Weill Cornell Medical Center, New York City. The goal is to increase awareness, collaboration and understanding of lung cancer, and to help participants implement the latest developments across the globe. The conference will cover a wide range of disciplines and unveil several research studies and clinical trial results. The global, single-arm, first-in-human ARROS-1 trial included a Phase II cohort of patients with locally advanced or metastatic ROS1-positive NSCLC who were naive to TKI therapy. Up to one prior line of chemotherapy and/or immunotherapy was permitted.

Original title: “Zidesamtinib Demonstrates 93% Response Rate in TKI-Naive Patients With Advanced ROS1-Positive NSCLC”

During clinical trials, severe infections reduced from 2 per year to 0.12-0.15 per year post-treatment, while moderate and severe bleeding episodes reduced from 1 to 0.16 per year. According to GlobalData’s Drugs database, Waskyra is just one of 24 cell and gene therapies (CGTs) that are currently approved for treating rare diseases ... During clinical trials, severe infections reduced from 2 per year to 0.12-0.15 per year post-treatment, while moderate and severe bleeding episodes reduced from 1 to 0.16 per year. According to GlobalData’s Drugs database, Waskyra is just one of 24 cell and gene therapies (CGTs) that are currently approved for treating rare diseases in the US. In clinical trials, 51% of patients were surgery free at year one. Of these patients, 14 out of 18 had still undergone zero surgeries after three years. There are now more than 1,500 cell and gene therapies for rare diseases in active clinical development globally. As the rise of CGTs drives the next stage of growth for orphan drugs, developers must confront the complexities of two of the most demanding areas of drug development in order to bring these advanced therapies to small, underserved patient populations. Despite the challenges, the landscape is evolving and maturing, with growing clinical pipelines and new product launches reshaping the outlook for rare disease treatment. Cell and gene therapies are fuelling new possibilities for rare diseases, turning a field defined by unmet need into one of the industry’s most dynamic areas of innovation.

Original title: “The rare disease revolution: The state of play for CGTs in orphan diseases - Pharmaceutical Technology”

During clinical trials, severe infections reduced from 2 per year to 0.12-0.15 per year post-treatment, while moderate and severe bleeding episodes reduced from 1 to 0.16 per year. According to GlobalData’s Drugs database, Waskyra is just one of 24 cell and gene therapies (CGTs) that are currently approved for treating rare diseases ... During clinical trials, severe infections reduced from 2 per year to 0.12-0.15 per year post-treatment, while moderate and severe bleeding episodes reduced from 1 to 0.16 per year. According to GlobalData’s Drugs database, Waskyra is just one of 24 cell and gene therapies (CGTs) that are currently approved for treating rare diseases in the US. In clinical trials, 51% of patients were surgery free at year one. Of these patients, 14 out of 18 had still undergone zero surgeries after three years. There are now more than 1,500 cell and gene therapies for rare diseases in active clinical development globally. As the rise of CGTs drives the next stage of growth for orphan drugs, developers must confront the complexities of two of the most demanding areas of drug development in order to bring these advanced therapies to small, underserved patient populations. Despite the challenges, the landscape is evolving and maturing, with growing clinical pipelines and new product launches reshaping the outlook for rare disease treatment. Cell and gene therapies are fuelling new possibilities for rare diseases, turning a field defined by unmet need into one of the industry’s most dynamic areas of innovation.

Original title: “The rare disease revolution: The state of play for CGTs in orphan diseases - Pharmaceutical Technology”

Phase 1 trial shows strong safety and CNS exposure

Original title: “Teitur reports positive early data for Parkinson’s peptide TT‑P34”