The Termeer Foundation’s Post

𝐒𝐩𝐫𝐢𝐧𝐠𝐖𝐨𝐫𝐤𝐬 𝐓𝐡𝐞𝐫𝐚𝐩𝐞𝐮𝐭𝐢𝐜𝐬 𝐈𝐧𝐢𝐭𝐢𝐚𝐭𝐞𝐬 𝐍𝐃𝐀 𝐒𝐮𝐛𝐦𝐢𝐬𝐬𝐢𝐨𝐧 𝐟𝐨𝐫 𝐌𝐢𝐫𝐝𝐚𝐦𝐞𝐭𝐢𝐧𝐢𝐛 𝐟𝐨𝐫 𝐍𝐅𝟏-𝐏𝐍 𝐓𝐫𝐞𝐚𝐭𝐦𝐞𝐧𝐭 SpringWorks Therapeutics (Nasdaq: SWTX) SpringWorks Therapeutics has commenced the rolling submission of a New Drug Application (NDA) to the FDA for mirdametinib, an investigational MEK inhibitor, targeting pediatric and adult patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN). Key Points: CEO Saqib Islam Saqib Islam highlights the potential of mirdametinib to address the unmet needs of NF1-PN patients. The rolling NDA submission marks progress towards providing a best-in-class therapy for these patients. Data from the Phase 2b ReNeu trial support the submission, showing promising response rates and improvements in patient-reported outcomes. Mirdametinib has received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the FDA and the European Commission. SpringWorks expects to complete the NDA submission by the second quarter of 2024. In summary, SpringWorks Therapeutics' NDA submission for mirdametinib represents a significant step forward in addressing NF1-PN, offering hope to patients and their families. #Mirdametinib #NF1 #Neurofibromatosis #RareDiseases #CancerTreatment #MEKInhibitor #FDAApproval #NDAsubmission #OrphanDrug #FastTrackDesignation #PediatricDisease

🚀 Exciting News Alert! 🚀   Cabaletta Bio has just received FDA Orphan Drug Designation for CABA-201, a promising therapy aimed at treating Systemic Sclerosis (SSc). This designation not only signifies a pivotal milestone for Cabaletta, but also holds immense significance for investors and potential acquirers. In addition to Cabaletta Bio's CABA-201, other emerging CAR-T therapies in the SSc space include Guangdong Ruishun Biotech's RJMty19, Kyverna Therapeutics' KYV-101, and Juno Therapeutics' BMS-986353.   ✅ Commercial Exclusivity: The ODD ensures a defined period of commercial exclusivity, providing assurance for investors and potential acquirers.   ✨ Potential Paradigm Shift: Cabaletta's rapid progress in advancing CAR T-cell therapy, especially in autoimmune diseases like SSc, and IIM, suggests a potential paradigm shift in treatment approaches for these conditions.   ⚡ Interim Results Awaited: The interim results from Phase 1/2 studies will be pivotal, serving as evidence to determine if CABA-201 truly has the potential to revolutionize treatment for autoimmune disorders. Source - https://lnkd.in/gHqbetW2 #CabalettaBio #AutoimmuneDisease #Innovation #Healthcare #ODD #InvestingOpportunity

Clinical Trials Updates: The FDA grants fast-track designation to Certa Therapeutics systemic sclerosis drug candidate. Great news for the treatment of systemic sclerosis! Certa Therapeutics has been granted fast-track designation by the FDA for their drug candidate, FT011. This chronic autoimmune disease causes abnormal connective tissue growth, leading to skin and organ hardening, with no known cure. Certa Therapeutics' phase II trial showed a 60% clinically significant improvement in patients compared to a placebo group. These results are impressive and have led to the drug being fast-tracked for regulatory evaluation, meaning it could reach the market faster. Congratulations to Certa Therapeutics for reaching this milestone and providing hope for those suffering from systemic sclerosis. #clinicaldevelopment #regulatoryaffairs #medicalaffairs #inflammation #sclerosis #FDA https://lnkd.in/e6_DXrGa

iRegene Receives IND approval from U.S. FDA to Start Clinical Trial for Parkinson’s Disease - https://lnkd.in/g6vekNF6 On June 20 (UTC-4), the U.S. Food and Drug Administration (FDA) approved the IND application for NouvNeu001, a cell therapy product from iRegene Therapeutics targeting Parkinson’s Disease. This marks a significant milestone as NouvNeu001 becomes the world’s first chemically induced allogeneic cell therapy product to enter clinical stages for Parkinson’s disease in the US. Previously, the “Combined Phase I/II Clinical Study” of NouvNeu001 had already received approval from China NMPA in August 2023 and demonstrated positive safety and efficacy data in its Phase I trials. On June 20, 2024, iRegene further announced IND approval of NouvNeu001 by the U.S. FDA for the treatment of mid-to-late stage of Parkinson’s disease. Read more at: https://lnkd.in/g6vekNF6 #parkinsonsdisease #regenerativemedicine #stemcell

Did you see the article by our COO Carol Woodward and Vice President of Regulatory Services Catherine Moncad in BioSpace?  In the US, an estimated 25 to 30 million people live with a rare disease. And yet, only 5% of these conditions have FDA-approved therapeutics.  Rare disease specialists and patients around the world desperately need options. The problem? These conditions don’t fit the mould, presenting several obstacles to straightforward drug development.  The main issue blocking these processes is small patient populations, argue Carol and Catherine.  Read more below…  #RareDiseases #CRO #ClinicalResearch  https://lnkd.in/eAhFYjPV 

🔍💡 We spoke with Rick Hawkins, CEO and Chairman of Lumos Pharma, to learn about the company’s patient-centric strategy for pediatric rare disease drug development and more. Read the full interview with Hawkins: https://buff.ly/3SyUZjs #RareDisease #PGHD

The Certa team is thrilled to announce that our candidate drug for the treatment of Scleroderma, FT011, has been granted Fast Track approval by the FDA, based on the positive Phase 2 data. This achievement is a significant milestone in our efforts to provide effective therapies that improve the lives of people living with #Scleroderma. 🟨 FT011 is a novel, first-in-class oral therapy for the treatment of chronic #fibrosis in multiple organs. 🟨 It targets an important but previously undrugged membrane GPCR receptor, #GPR68. FT011 has the potential to revolutionise the treatment of Scleroderma, and with the Fast Track approval, we are one step closer to offering a therapy that can go beyond treating the symptoms. Professor Darren Kelly, Certa Therapeutics CEO and founder, “𝘞𝘦 𝘢𝘳𝘦 𝘵𝘩𝘳𝘪𝘭𝘭𝘦𝘥 𝘵𝘰 𝘩𝘢𝘷𝘦 𝘳𝘦𝘤𝘦𝘪𝘷𝘦𝘥 𝘍𝘢𝘴𝘵 𝘛𝘳𝘢𝘤𝘬 𝘋𝘦𝘴𝘪𝘨𝘯𝘢𝘵𝘪𝘰𝘯, 𝘸𝘩𝘪𝘤𝘩 𝘴𝘶𝘱𝘱𝘰𝘳𝘵𝘴 𝘧𝘶𝘳𝘵𝘩𝘦𝘳 𝘢𝘤𝘤𝘦𝘭𝘦𝘳𝘢𝘵𝘪𝘰𝘯 𝘰𝘧 𝘵𝘩𝘦 𝘍𝘛011 𝘤𝘭𝘪𝘯𝘪𝘤𝘢𝘭 𝘥𝘦𝘷𝘦𝘭𝘰𝘱𝘮𝘦𝘯𝘵 𝘱𝘳𝘰𝘨𝘳𝘢𝘮. 𝘐𝘵 𝘪𝘴 𝘢𝘭𝘴𝘰 𝘱𝘳𝘰𝘷𝘪𝘥𝘪𝘯𝘨 𝘷𝘢𝘭𝘪𝘥𝘢𝘵𝘪𝘰𝘯 𝘰𝘧 𝘍𝘛011’𝘴 𝘱𝘰𝘵𝘦𝘯𝘵𝘪𝘢𝘭 𝘵𝘰 𝘰𝘧𝘧𝘦𝘳 𝘱𝘢𝘵𝘪𝘦𝘯𝘵𝘴 𝘸𝘪𝘵𝘩 𝘴𝘤𝘭𝘦𝘳𝘰𝘥𝘦𝘳𝘮𝘢 𝘵𝘩𝘦 𝘧𝘪𝘳𝘴𝘵 𝘢𝘯𝘵𝘪-𝘧𝘪𝘣𝘳𝘰𝘵𝘪𝘤 𝘢𝘯𝘥 𝘥𝘪𝘴𝘦𝘢𝘴𝘦-𝘮𝘰𝘥𝘪𝘧𝘺𝘪𝘯𝘨 𝘵𝘳𝘦𝘢𝘵𝘮𝘦𝘯𝘵 𝘰𝘧 𝘵𝘩𝘪𝘴 𝘵𝘺𝘱𝘦.” “𝘞𝘦 𝘬𝘯𝘰𝘸 𝘵𝘩𝘢𝘵 𝘵𝘩𝘪𝘴 𝘥𝘦𝘣𝘪𝘭𝘪𝘵𝘢𝘵𝘪𝘯𝘨 𝘢𝘯𝘥 𝘭𝘪𝘧𝘦-𝘵𝘩𝘳𝘦𝘢𝘵𝘦𝘯𝘪𝘯𝘨 𝘥𝘪𝘴𝘦𝘢𝘴𝘦 𝘤𝘢𝘯 𝘴𝘦𝘷𝘦𝘳𝘦𝘭𝘺 𝘪𝘮𝘱𝘢𝘤𝘵 𝘵𝘩𝘦 𝘭𝘪𝘷𝘦𝘴 𝘰𝘧 𝘱𝘢𝘵𝘪𝘦𝘯𝘵𝘴, 𝘢𝘯𝘥 𝘵𝘰 𝘥𝘢𝘵𝘦, 𝘦𝘹𝘪𝘴𝘵𝘪𝘯𝘨 𝘵𝘳𝘦𝘢𝘵𝘮𝘦𝘯𝘵𝘴 𝘰𝘯𝘭𝘺 𝘧𝘰𝘤𝘶𝘴 𝘰𝘯 𝘵𝘩𝘦 𝘳𝘦𝘭𝘪𝘦𝘧 𝘢𝘯𝘥 𝘮𝘢𝘯𝘢𝘨𝘦𝘮𝘦𝘯𝘵 𝘰𝘧 𝘴𝘺𝘮𝘱𝘵𝘰𝘮𝘴, 𝘸𝘩𝘦𝘳𝘦𝘢𝘴 𝘍𝘛011 𝘱𝘳𝘦𝘤𝘪𝘴𝘦𝘭𝘺 𝘵𝘢𝘳𝘨𝘦𝘵𝘴 𝘵𝘩𝘦 𝘳𝘰𝘰𝘵 𝘤𝘢𝘶𝘴𝘦 𝘰𝘧 𝘧𝘪𝘣𝘳𝘰𝘴𝘪𝘴 𝘢𝘯𝘥 𝘩𝘢𝘴 𝘵𝘩𝘦 𝘱𝘰𝘵𝘦𝘯𝘵𝘪𝘢𝘭 𝘵𝘰 𝘰𝘧𝘧𝘦𝘳 𝘵𝘳𝘦𝘢𝘵𝘮𝘦𝘯𝘵 𝘢𝘤𝘳𝘰𝘴𝘴 𝘮𝘶𝘭𝘵𝘪𝘱𝘭𝘦 𝘰𝘳𝘨𝘢𝘯𝘴 𝘸𝘪𝘵𝘩𝘪𝘯 𝘵𝘩𝘦𝘴𝘦 𝘱𝘢𝘵𝘪𝘦𝘯𝘵𝘴.” #drugdevelopment

Today, we announced that the U.S. Food and Drug Administration has cleared our Investigational New Drug (IND) application for CTIM-76, a Claudin 6 (CLDN6) x CD3 T cell engaging bispecific antibody. The IND supports the initiation of a Phase 1 dose escalation and expansion #clinicaltrial of CTIM-76 in patients with CLDN6-positive #gynecologic and #testicular #cancers. Context anticipates the enrollment of the first patient in the dose escalation portion of its clinical trial in mid-2024. https://lnkd.in/g9FkTBNP

🌟 HIRC Insight Series 🌟 It is very encouraging to see the FDA's commitment to advancing treatments for rare diseases. This initiative marks a significant step forward in developing potentially life-changing therapies for patients with rare conditions. The next step is getting these treatments to the patients who need them. HIRC's new research with payers and specialty pharmacies assists manufacturers’ commercialization and market access efforts pertaining to rare disease portfolios. For more information visit: https://lnkd.in/gzUDFA4j #HIRCInsightSeries #HIRC #RareDisease #FDA #ClinicalTrials #STARTProgram #InnovativeTherapies #HealthcareAdvancement #Pharma #PharmaTrends #Pharmaceuticals #PharmaIndustry #PharmaNews #PharmaData #PharmaResearch #Healthcare #LifeSciences #Specialtypharmacy #Specialtypharmaceuticals #Orphandrugs #Genetherapy

The FDA is encouraging the inclusion of patient-reported outcomes (PROs) in cancer drug clinical trials to gain valuable insights into the patient experience without investigator bias. Studies reveal a lack of PRO assessments in trials leading to FDA approvals, impacting treatment decisions. PRO evidence can influence formulary reviews and provide context for therapy safety. Efforts are being made to improve PRO data collection and reporting in clinical trials, emphasizing the patient's voice in drug development and evaluation. Despite the benefits of PROs, their inclusion should