i&i Biotech Fund’s Post

“Transforming Lives of Cystic Fibrosis Patients: A New Era for Orphan Drugs 🌟” “Cystic fibrosis is one of the rare diseases that has challenged the medical community for decades. However, with the introduction of breakthrough treatments like Trikafta, hope is now closer than ever. This revolutionary therapy addresses the root cause of the disease, leading to significant improvements in lung function and quality of life for over 90% of eligible patients. Advancements in orphan drugs are not just scientific milestones but also messages of hope for millions of patients worldwide who silently endure the challenges of rare diseases. How can we encourage more investment in scientific research to provide innovative solutions for these underserved patient populations? Let’s discuss!” #CysticFibrosis #OrphanDrugs #HealthcareInnovation #ScientificResearch# vertex pharmaceutical

Looking to advance your professional development? Register for this PACE-accredited webinar. Experts Prof. Dr. Hatice Bodur and Dr. Gülsen Yılmaz explore the clinical utility of therapeutic drug monitoring (TDM) for TNFa inhibitors such as adalimumab and infliximab. #arthiritis #clinicalwebinar #clinicalscience #TDM

✨ Happy New Year 2025!! May this year bring all of you happiness, success and health! 💊 ENYO Pharma is deeply mobilized to help patients suffering from Chronic Kidney Diseases by advancing its portfolio of #antiinflammatory and #fibrolytic drugs, especially Vonafexor for Alport syndrome patients. 👨⚕️ ENYO Pharma is moving forward rapidly with the enrollment of Alport syndrome patients in our ALPESTRIA-1 Phase 2 clinical study with Vonafexor. The last-patient-in will start to be dosed in the coming days. #biotech #pharma #CKD #kidneydisease #raredisease #clinicaltrial

🎉 Exciting News! I'm thrilled to share that Lmito Therapeutics has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for LMT801, our investigational treatment for Charcot-Marie-Tooth Disease Type 1A (CMT1A). CMT1A is a rare genetic disorder affecting approximately 1 in 2,500 people worldwide, with about 126,000 patients in the U.S. and 3 million globally. Currently, there are no approved treatments for this devastating condition. Key highlights about LMT801: - Demonstrated significant improvements in motor function, sensory function, and nerve conduction in CMT1A animal models - Showed excellent safety profile in long-term oral administration studies - Potential to be a first-in-class therapy for CMT1A   The ODD grant provides several development incentives, including: Tax credits for qualified clinical trials, Exemption from user fees, Potential 7-year market exclusivity upon approval. #RareDiseases #Biotech #DrugDevelopment #Innovation #Healthcare #FDA #OrphanDrug #CMT #Research

Seeking Participants for a New CMT Clinical Trial (for the US only):  A Chance to Help Advance Treatment! The sponsor of the study is NMD Pharma A/S. If you’re living with Charcot-Marie-Tooth (CMT) disease, specifically CMT1 or CMT2 subtypes, you may be eligible to participate in an exciting new clinical trial.  NMD Pharma, a biotechnology company, has received clearance from the U.S. Food and Drug Administration (FDA) to proceed with the SYNAPSE-CMT Phase 2 trial. This trial will evaluate the safety, tolerability, and potential effectiveness of an investigational medication, NMD670, which could improve muscle strength and endurance in individuals with CMT. What’s the Goal of the Study? NMD670 may enhance communication between nerves and muscles where transmission is impaired, a common issue for people living with CMT. The hope is that this treatment will improve your strength, endurance, and overall daily functioning. By joining the study, you’ll play a critical role in determining whether this therapy could help others with CMT in the future. Learn more or to participate:  https://lnkd.in/eYSKTrKa https://lnkd.in/eHsEZ6MP #NMDpharma #cmtawareness #hnf4cmt #clinicaltrials #cmtresearch #charcotmarietooth #cmtneuropathy #cmtgene #charcotmarietoothdisorder #fda

Pathalys pharma secures $105 million series B financing. Oversubscribed financing paves path to NDA submission and pre-commercialization efforts for upacicalcet.  Upacicalcet is a novel calcimimetic that can be delivered intravenously at the end of the hemodialysis session via pre-filled syringe and has been demonstrated to effectively reduce levels of parathyroid hormone(PTH) and may have a better tolerability profile for patients. Upacicalcet can be used in secondary hyperparathyroidism (SHPT) to address an unmet need in the management of advanced chronic kidney disease (CKD). Chemenu has been working to develop more compounds for drug discovery. Here comes the building blocks we can provide: https://lnkd.in/gsg24G8S Keywords: Upacicalcet; CaSR; secondary hyperparathyroidism (SHPT); building blocks  

Catalyst to watch out in July 2024 Worried that you might lose track of major clinical developments? Octavus Consulting has got you covered! With our Monthly Catalyst, we keep you updated on key drug candidates with PDUFA Events and NDA Acceptance. Mark your calendars for: 1. ZORYVE by Arcutis Biotherapeutics for Atopic Dermatitis (6yrs ≥) 2. OX124 by Orexo for Opioid Overdose 3. VOQUENZA by Phathom Pharmaceuticals for Heartburn Associated with Nonerosive GERD 4. ALPHA-1062 by Alpha Cognition for Mild to Moderate Alzheimer’s Disease 5. DASYNOC by Xspray Pharma for Chronic Myeloid Leukemia (CML) Stay up to date on major drug developments with Octavus Consulting! Contact us at bd@octavusconsulting.com for: • Competitor Monitoring • Competitive Landscape Analysis • Clinical Trial Insights • Competitor Profiling Stay tuned on LinkedIn for industry-related insights and updates! Together, let’s navigate biopharma innovation! Arcutis Biotherapeutics, Inc. (Nasdaq: ARQT) Orexo US, Inc. Phathom Pharmaceuticals Alpha Cognition, Inc. XSPRAY PHARMA AB (PUBL) #fda #pdufa #drugdevelopment #biotech #biopharma #pharma #healthcare #biotechindustry #zoryve #ox124 #voquenza #alpha1062, #dasynoc

Yesterday's FDA approval of Eli Lilly's Kisunla for the treatment of early symptomatic Alzheimer's disease was the second FDA approval for the treatment of the Alzheimer's. Find out which other drugs are targeting the degenerative disease with our drug & pipeline screener tool. #fasterscience #biopharmcatalyst #catalysts #biotechinvesting #pharma #biotechstocks #alzheimers

Don't loose track of your competition and your peers. Stay ahead with Octavus Consulting's monthly tracking major events This month mark your calendars for: 1. ZORYVE by Arcutis Biotherapeutics, Inc. (Nasdaq: ARQT) for Atopic Dermatitis (≥6yrs.) 2. OX124 by Orexo US, Inc. for Opioid Overdose 3. VOQUENZA by Phathom Pharmaceuticals for Heartburn Associated with Nonerosive GERD 4. ALPHA-1062 by Alpha Cognition, Inc. for Mild to Moderate Alzheimer’s Disease 5. DASYNOC by XSPRAY PHARMA AB (PUBL) for Chronic Myeloid Leukemia (CML) Stay up to date on major drug developments with Octavus Consulting! Contact us at bd@octavusconsulting.com for: • Competitor Monitoring • Competitive Landscape Analysis • Clinical Trial Insights • Competitor Profiling • BD&L monitoring and tracking Stay tuned on LinkedIn for industry-related insights and updates! Together, let’s navigate biopharma innovation! #PDUFA #FDA FDA #Approval #tracking #competitoranalysis

Today at #ESMOBreast24 we shared some of the latest clinical data for our investigational PROTAC ER degrader being developed jointly with Pfizer as part of a potential combination therapy for patients with ER+/HER2- advanced or metastatic #BreastCancer. We are encouraged by this updated clinical data in this Phase 1b study and the potential this novel therapy may offer people with breast cancer. #BCSM