Alligator Bioscience's OPTIMIZE-1 trial and FDA's Project Optimus

FDA lifts hold on Neurizon's ALS asset, clearing path for Phase II/III trials FDA has officially lifted the clinical hold on Neurizon Therapeutics’ lead candidate NUZ-001, clearing the way for its Phase 2/3 inclusion in the HEALEY ALS Platform Trial—expected to begin Q4 CY2025. With the IND now active, Neurizon anticipates Mass General Hospital (MGH) will soon file a protocol amendment to incorporate the NUZ-001 regimen into the trial. Patient enrollment is targeted to start later this year. This regulatory milestone establishes a validated IND framework, accelerating development not only for NUZ-001 but also for future pipeline programs—streamlining safety, manufacturing, and design standards to reduce regulatory risk and shorten timelines. #ALS #NeurizonTherapeutics #NUZ001 #HEALEYTrial #FDA #ClinicalTrials #DrugDevelopment #Neurodegeneration #Biotech #RegulatoryMilestone https://lnkd.in/gxFUMuzu

Vasa Therapeutics secures FDA IND clearance to advance VS-041 into clinical trial for HFpEF https://lnkd.in/gZwwpTbY Vasa Therapeutics #biotechnology #noveltherapies #cardiovascular #metabolicaging U.S. Food and Drug Administration - Office of Criminal Investigations (OCI) 

Brian Windsor, Chief Executive Officer of Rein Therapeutics, commented, “These new authorizations mark another important milestone in our global RENEW trial. With approvals now in the U.K., Germany, and Poland, we are well positioned to begin enrolling patients in multiple regions and advance LTI-03 toward our goal of redefining how pulmonary fibrosis is treated. We are grateful to our clinical partners across Europe for their collaboration and commitment to improving outcomes for patients with IPF.” #Biotech #ClinicalTrials #Immunotherapy #PulmonaryFibrosis #IPF #DrugDevelopment #ReinTherapeutics #LTI03 #MedicalInnovation #Pharma https://lnkd.in/dmka3xGV

Applied Therapeutics, Inc. (NASDAQ: $APLT) rallies after posting strong results from a confirmatory study—raising hopes of alignment with FDA for its rare disease drug. https://lnkd.in/erpfQxsV

Exciting and important news from our portfolio company Gesynta Pharma that today announced that its clinical trial application for the company's Phase 2 trial of vipoglanstat for the treatment of endometriosis has been approved by the UK authorities. https://lnkd.in/dTcKpVva

Over the past 25–30 years, there has been a significant increase in the approval of new molecular entities (NMEs) by the FDA for the treatment of oncological and hematological malignancies. Between 1998 and the end of 2024, a total of 217 NMEs were approved, with the average annual approval rate increasing from 3.5 during the 1998-2010 period to 12.2 between 2011 and 2024. A substantial proportion of these NMEs are targeted therapies, with 78 (36%) associated with companion diagnostic (CDx) assays. Kinase inhibitors were most frequently linked to CDx assays (60%), followed by antibodies (39%) and small-molecule drugs (26%). The proportion of NMEs approved with a CDx assay rose from 15% during 1998-2010 to 42% in 2011–2024. Notably, nine (4%) NMEs were approved for tissue-agnostic indications, for which the CDx assay is critical for patient stratification. However, the data indicate that the approval of a CDx assay does not always coincide with the approval of the corresponding tumor-agnostic drug. The number of NMEs with associated CDx assays is expected to increase as innovative molecular analytical technologies, such as RNA sequencing and mass spectrometry, are incorporated as platforms for CDx assays. This article is open access: https://lnkd.in/dwBSNaca #medicine #pharmaceuticals #pharma #genomics #oncology #precisionmedicine

The Sanofi partnership will support clinical validation and regulatory submissions for Revvity’s new assay. Elsewhere, Kihealth raised $5 million in seed funding to launch its early detection test.

Exciting news from the FDA as they announce the distribution of the first nine national priority vouchers, benefiting companies like Merck KGaA, Regeneron, and Sanofi. Introduced by the FDA Commissioner in June, and open for applications since July, the priority voucher program aims to expedite the approval process for medications aligning with U.S. national priorities. Historically, this process takes 10 to 12 months, but with the program's focus on addressing public health challenges, meeting significant medical needs, and providing groundbreaking treatments, companies can now expect approval in just one to two months. The Commissioner's National Priority Voucher (CNPV) recipients consist of both approved drugs and investigational medicines, reflecting a diverse range of advancements in the medical field. https://lnkd.in/dcD_RFgu

💊 This week in #pharma: Big deals, bold data, and a boost for UK pricing policy. From multibillion-dollar acquisitions to policy shifts and pipeline progress, this week’s #FridayFive highlights the top developments shaping the industry: 1️⃣ Novo Nordisk adds to #MASH momentum with its $4.7B Akero buyout, expanding its FGF21 and metabolic disease pipeline. 2️⃣ AstraZeneca’s baxdrostat delivers another Phase III win in treatment-resistant #hypertension. 3️⃣ Peter Marks, former FDA biologics chief, takes on a new role at Eli Lilly and Company leading molecule discovery. 4️⃣ UK government considers raising NICE - National Institute for Health and Care Excellence cost-effectiveness thresholds to attract pharma investment. 5️⃣ AstraZeneca and Daiichi Sankyo US’s Datroway shows strong results in triple-negative #breastcancer, setting up a potential frontline shift. Read the full rundown 👉 https://lnkd.in/dmdEYeSE #ADCs #clinicalresearch #clinicaltrials #drugdevelopment #FDA #medicalaffairs #mergersandacquisitions #oncology #pharmaceuticals

Novo Nordisk Strikes $2.1 Billion Deal with Omeros, Expanding Rare Disease Portfolio – ( $NVO $OMER $IBB $XBI ) https://lnkd.in/gp6SyqFj #NovoNordisk #Omeros #biotech #pharma #pharmaceuticals #rareDisease #drugdevelopment #assetdeal #biotechnews #stockmarket #clinicaltrials #MASP3 #zaltenibart #healthcare #biopharma #licensingdeal #acquisition #medicalinnovation #blooddisorders #renaldisorders #PNH #marketupdate #stockperformance #industrynews #drugpipeline #research