X4 Pharmaceuticals’ Post

This morning MC2 Therapeutics announced positive feedback from our pre-IND meeting with the FDA concerning our phase 2 stage first-in-class oral drug candidate, MC2-32. Such interim regulatory alignment and guidance are instrumental - especially as we are pioneering uncharted territory in medical science to help people with safe and effective novel treatment paradigms. We also announced that pre-clinical studies have demonstrated that oral MC2-32 not only holds promise for our lead indication Hidradenitis Suppurativa but also for other challenging conditions like neutrophilic dermatoses and beyond skin conditions. Looking ahead, MC2 is on track to file an IND for a Phase 2b clinical trial with MC2-32 in Hidradenitis Suppurativa. We're not stopping there – we also plan to initiate a Phase 2 trial for Pyoderma Gangrenosum, a rare and painful ulcerative condition with currently no approved treatments, as part of our broader strategy to tackle additional underserved diseases with MC2-32 within Immunology and Inflammation. This progress is another testament to the exceptional dedication and skills of our MC2 Therapeutics Team - Thank you! Read the full announcement here https://lnkd.in/dtbbz3H6 #MC2Therapeutics #HealthcareInnovation #Hidradenitissuppurativa #Biotechnology #pyodermagangrenosum #Immunology #Inflammation

An very important step forward - for MC2 and for patients suffering from Hidradenitis Suppurativa. Now potentially also for patients with Pyoderma Gangrenosum.

We are pleased to announce that we have received positive feedback from our pre-IND meeting with the FDA and are on track to file an IND in mid-2025 for a Phase 2b clinical trial in Hidradenitis Suppurativa (#HS) with MC2-32, our first-in-class oral drug candidate targeting multiple pathways involved in I&I indications.   Given MC2-32’s unique mode of action and pharmacological profile it is well positioned to address a multitude of other neutrophilic dermatoses as well as other large and rare I&I diseases which is currently being explored pre-clinically by MC2. MC2 Therapeutics is planning to explore MC2-32 in additional Phase 2 trials starting in 2025 with Pyoderma Gangrenosum (#PG), a rare skin condition causing painful ulcers, for which there is a significant unmet patient need and no approved treatments.   Read the full announcement for more on our indication expansion strategy: https://lnkd.in/dGNc39Fa   #MC2Therapeutics #Immunology #Inflammation #Biotech #Innovation #ClinicalResearch 

Explore the rapidly evolving landscape of immunology in the latest #IQVIA blog as we delve into the future #immunology, highlighting untapped opportunities in smaller immunology indications. Our author Markus Gores emphasizes that innovators must focus on patient-relevant evidence and close collaboration with #patient advocacy groups to see success. Read now for more insights: https://bit.ly/44ckues

📢 News from MHRA! 📢 The Medicines and Healthcare products Regulatory Agency (MHRA) has approved the use of capivasertib (Truqap) for patients with advanced hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative breast cancer that has one or more abnormal PIK3CA, AKT1, or PTEN genes and does not respond to other anti-hormonal therapies. Capivasertib, an AKT inhibitor, works by blocking ATK Kinases, proteins that enable cancer cell growth and multiplication. This mechanism helps reduce the growth and spread of advanced breast cancer and aids in destroying cancer cells. Capivasertib is administered alongside fulvestrant, a hormonal therapy, at a starting dose of 400 mg twice a day for four days, followed by three days of rest, in a repeating cycle. The approval is based on a clinical trial involving 708 patients with locally advanced or metastatic HR-positive, HER2-negative breast cancer, including 289 with abnormal PIK3CA, AKT1, or PTEN genes. Patients receiving capivasertib experienced an average of 7.3 months without cancer progression compared to 3.1 months for those on a placebo. Common side effects include high blood sugar, diarrhoea, rash, urinary tract infection, low haemoglobin, loss of appetite, nausea, vomiting, mouth sores, itching, and tiredness. The MHRA will continuously monitor the safety and effectiveness of Truqap. Patients experiencing side effects should report them through the Yellow Card scheme. 🔗 For more information or to report side effects, visit: https://lnkd.in/dCKtSMWF #MHRA #Healthcare #Pharmaceuticals #BreastCancer #Capivasertib #Truqap #CancerTreatment #ClinicalTrials #MedicalNews #PatientCare #HealthInnovation

📢 News from MHRA! 📢 The Medicines and Healthcare products Regulatory Agency (MHRA) has approved the use of capivasertib (Truqap) for patients with advanced hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative breast cancer that has one or more abnormal PIK3CA, AKT1, or PTEN genes and does not respond to other anti-hormonal therapies. Capivasertib, an AKT inhibitor, works by blocking ATK Kinases, proteins that enable cancer cell growth and multiplication. This mechanism helps reduce the growth and spread of advanced breast cancer and aids in destroying cancer cells. Capivasertib is administered alongside fulvestrant, a hormonal therapy, at a starting dose of 400 mg twice a day for four days, followed by three days of rest, in a repeating cycle. The approval is based on a clinical trial involving 708 patients with locally advanced or metastatic HR-positive, HER2-negative breast cancer, including 289 with abnormal PIK3CA, AKT1, or PTEN genes. Patients receiving capivasertib experienced an average of 7.3 months without cancer progression compared to 3.1 months for those on a placebo. Common side effects include high blood sugar, diarrhoea, rash, urinary tract infection, low haemoglobin, loss of appetite, nausea, vomiting, mouth sores, itching, and tiredness. The MHRA will continuously monitor the safety and effectiveness of Truqap. Patients experiencing side effects should report them through the Yellow Card scheme. 🔗 For more information or to report side effects, visit: https://lnkd.in/dCKtSMWF #MHRA #Healthcare #Pharmaceuticals #BreastCancer #Capivasertib #Truqap #CancerTreatment #ClinicalTrials #MedicalNews #PatientCare #HealthInnovation

📢 News from MHRA! 📢 The Medicines and Healthcare products Regulatory Agency (MHRA) has approved the use of capivasertib (Truqap) for patients with advanced hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative breast cancer that has one or more abnormal PIK3CA, AKT1, or PTEN genes and does not respond to other anti-hormonal therapies. Capivasertib, an AKT inhibitor, works by blocking ATK Kinases, proteins that enable cancer cell growth and multiplication. This mechanism helps reduce the growth and spread of advanced breast cancer and aids in destroying cancer cells. Capivasertib is administered alongside fulvestrant, a hormonal therapy, at a starting dose of 400 mg twice a day for four days, followed by three days of rest, in a repeating cycle. The approval is based on a clinical trial involving 708 patients with locally advanced or metastatic HR-positive, HER2-negative breast cancer, including 289 with abnormal PIK3CA, AKT1, or PTEN genes. Patients receiving capivasertib experienced an average of 7.3 months without cancer progression compared to 3.1 months for those on a placebo. Common side effects include high blood sugar, diarrhoea, rash, urinary tract infection, low haemoglobin, loss of appetite, nausea, vomiting, mouth sores, itching, and tiredness. The MHRA will continuously monitor the safety and effectiveness of Truqap. Patients experiencing side effects should report them through the Yellow Card scheme. 🔗 For more information or to report side effects, visit: https://lnkd.in/dCKtSMWF #MHRA #Healthcare #Pharmaceuticals #BreastCancer #Capivasertib #Truqap #CancerTreatment #ClinicalTrials #MedicalNews #PatientCare #HealthInnovation

Another trip to Boston? ✈ It can only be for one event - the ultimate forum for mRNA dedicated biopharma stakeholders - The mRNA-Based Therapeutics Summit USA! What's more, with Moderna's latest RSV vaccine approval, there is immense excitment as we discuss the route to successful approval with none-other than the leading regulatory bodies and key regulatory change-makers that are making history as we speak! Join Dr Peter Marks (CBER, FDA), Ka-Wai Wan (Medicines and Healthcare products Regulatory Agency), Marco Cavaleri (European Medicines Agency), Carla Vinals (Moderna), Nitisha Pyndiah, PhD (GSK), and Emily English (Cartesian Therapeutics) as they discuss the following questions: 🔹 How can we work towards successful regulatory approvals for mRNA technology as a platform to reduce the process for individual mRNA medicine applications and expedite to patients faster? 🔹 What steps can the industry take to help establish a regulatory framework around mRNA technology and combat all diseases? With 65+ key speakers and 40+ insightful case studies to share across infectious disease, oncology, rare disease and beyond, view the full program and registration details here: https://ter.li/z28m60 #mRNA #RNA #mRNATherapeutics #mRNAVaccines #WorldRNA

Viridian Therapeutics, Inc. reaches a milestone by completing enrollment in its phase 3 clinical trials, THRIVE and THRIVE-2, dedicated to patients with thyroid eye disease (TED). THRIVE-2, aimed at chronic TED, exceeded its enrollment goal, with 188 participants, 40% from US sites. Topline data is expected by the end of 2024. THRIVE, for active TED, surpassed its target in March 2024, with topline data anticipated in September 2024. Viridian's lead candidate, VRDN-001, is an intravenous monoclonal antibody showing promise in improving TED symptoms. The trials compare VRDN-001 to a placebo, offering a more efficient dosing regimen. Subcutaneous VRDN-003 trials, REVEAL-1 and REVEAL-2, are set to begin in August. The successful enrollment highlights the demand for TED treatment options, and Viridian is optimistic about addressing patient needs. Learn more: https://lnkd.in/dTrreZvr Laura A Epelbaum 🦁 Kirk Bertelsen Sarah Jane Constantine #Healthcare #ClinicalTrials #ThyroidEyeDisease

2024 is set to be a pivotal year for MC2 Therapeutics with significant milestones in our three Phase 2 stage clinical programs within Immunology and Inflammation. Next week in San Francisco, I am representing our dedicated MC2 Therapeutics team at the J.P. Morgan Healthcare Conference to provide an update on our progress and plans and to discuss collaborations with investors and industry partners.   Our two novel drug candidates (an oral HSP90 Inhibitor and a topical Iso-cyanate scavenger) are first-in-class with new mode of action and multiple indications potential, including Hidradenitis Suppurativa, CKD-associated Pruritus (CKD-aP) and Vulvar Lichen Sclerosus. Addressing the unmet needs of millions of patients and a testament to our commitment to pioneer novel treatment paradigms within I&I.   I look forward to engaging.   #JPM2024 #HealthcareInnovation #MC2Therapeutics #ClinicalAdvancement #Networking