CTI Life Sciences Fund’s Post

Medicus Pharma Ltd. (TSX-V:MDCX) announced that it has submitted a Phase 2 Investigational New #Drug (IND) clinical protocol to non-invasively treat basal cell carcinoma (BCC) of the skin using micro-array needles containing doxorubicin (D-MNA) developed by its portfolio company SkinJect to the United States Food and Drug Administration (FDA). The company said it is seeking feedback from the FDA on the IND to finalize the protocol. It said the study, SKNJCT-003, will enroll up to 60 patients presenting with nodular type BCC of the skin and will evaluate the efficacy of two dose levels of D-MNA when compared with a placebo. The low-dose group will receive 100μg of D-MNA and the high-dose group 200μg of D-MNA, the maximum tolerated dose established in SkinJect’s Phase 1 safety and tolerability trial carried out in March 2021. More at #Proactive #ProactiveInvestors http://ow.ly/810M1057Hvy #TSXV #MDCX

Milestone Achieved: FDA Greenlights Gilead's Newly Poised “Livdelzi” to Make a Big Impact Perhaps you caught our previous LinkedIn post that was shared by us 2 months ago. As it happens, what we’ve been hoping for has come to fruition. Gilead Sciences has now received FDA approval for Livdelzi (seladelpar), a new treatment for a rare liver disease known as primary biliary cholangitis. This approval is crucial for patients who can’t tolerate or don't respond well to existing treatments. Livdelzi is now available alone or in combination with other therapies and has strong potential in the market. How will Livdelzi's FDA approval reshape the options available for patients with primary biliary cholangitis? RBC Capital Markets analyst Brian Abrahams noted that Livdelzi's "cleaner label and itch relief benefits" are likely to give it a competitive advantage. Feel free to check out the article linked in the comments below.   #Livdelzi #FDAApproval #PrimaryBiliaryCholangitis #GileadSciences #Biotech #Pharma #HealthcareInnovation #LiverDisease #NewTreatment #CymaBayTherapeutics #DrugDevelopment #MarketImpact #MedicalBreakthrough

OBEFAZIMOD, A FIRST-IN-CLASS DRUG FOR THE TREATMENT OF ULCERATIVE COLITIS   Abivax Reports: Positive Interim Efficacy and Safety Analysis of Once-Daily 25mg Obefazimod in Moderate to Severe Ulcerative Colitis Patients After 2-Years of Open-Label Maintenance. Obefazimod (ABX464), Abivax’s lead investigational drug candidate, is an orally available small molecule with anti-inflammatory properties through the specific and selective upregulation of miR-124 expression. Phase 2 clinical trials in patients with UC have generated positive data, resulting in the initiation of a pivotal global Phase 3 clinical trial program (ABTECT Program), with first patients enrolled in the United States in October 2022. Initiation of a Phase 2b clinical trial in Crohn’s disease is expected in Q4 2024, and exploration of potential combination therapy opportunities in UC is ongoing. #Obefazimod #ABX464 #UlcerativeColitis   https://lnkd.in/d-MTMJD7

If you're looking to get an in-depth perspective on #psychedelics with a group of the top scientific experts in the industry, this is your conference. I've loved attending the past few years, and this is one of the few events in the sector where I can say I always learn something new. More details below!

That’s not the message of the study, Endpoints News—and it’s misleading at best. Here’s the real context: • “Only” 7% of RPDs led to PRVs because most RPDs are for either preclinical programs or programs a company hasn’t yet generated their own clinical data for • This is almost perfectly in line with standard success rates for early stage programs—not a shortcoming of the RPD/PRV program as is implied in the article • More relevant takeaways include the increasing frequency of RPDs being submitted alongside Fast Track and Orphan Drug Designations (implying RPDs are becoming a more integrated part of orphan drug development), the breadth of therapeutic areas and diseases receiving RPD, and the fact that at an average sale price of ~$100m, the 38 issued PRVs have the potential to generate $3.8 BILLION in funding for pediatric rare disease drug developers! #raredisease #priorityreviewvoucher #orphandrugs https://lnkd.in/dPqrJV9z

【NEWS UPDATE】Exciting news for the myasthenia gravis (gMG) community! 🎉 China's NMPA has approved ZaiLab's new biological product, Edgar moder - a subcutaneous injection dosage form to treat adult gMG patients with AChR antibodies. 💉 This novel FcRn inhibitor drug, licensed from Argenx, has demonstrated comparable efficacy to intravenous forms, with improved safety. 👨⚕️ This new treatment option will provide more flexibility in managing this autoimmune disorder and significantly improve patients' quality of life. 🙌 A true breakthrough in the field of antibody-mediated autoimmune diseases. #MG #Autoimmune #DrugApproval #FcRnInhibitor #ZaiLab https://lnkd.in/g6mfRUnD

In case you missed it, the European Commission granted orphan drug designation to our treatment for eosinophilic granulomatosis with polyangiitis (EGPA). Read the article from Pharmaceutical-Technology to learn more. #Pulmonology #RareDisease

New Novo Nordisk Hemophilia Drug Shows Promising Results in Phase 3 Trial Novo Nordisk's experimental hemophilia A treatment, Mim8, has demonstrated positive outcomes in its Phase 3 FRONTIER-2 trial, effectively controlling bleeding. Among patients with prior preventive treatments, 66% on once-weekly and 65% on once-monthly dosing achieved zero bleeds. Results showed the drug reduced the average annualized bleeding rate by 97% for once-weekly dosing, and 99% for once-monthly dosing for previously untreated patients - with no serious side effects reported. Novo Nordisk's Head of Development, Martin Holst Lange, emphasized the potential of Mim8 to offer patients the prospect of zero bleeds with convenient dosing flexibility. The company plans to submit an approval application for Mim8 by the end of the year. The OxyDial team is excited to see Mim8's promising results in treating patients with hemophilia A. We fully support the development of innovative new therapies that can provide significant life improvements through more flexible dosing options and better bleeding control. Read more: https://lnkd.in/efa96Z_D #Mim8 #HemophiliaA #ClinicalTrials #NovoNordisk #FDA #RareDisease

Yesterday was a great day for patients with NASH (now also known as MASH)! 🎊 Firstly, #MadrigalPharmaceuticals announced that their drug #Resmetirom has been approved by the FDA for the treatment of NASH patients in stages F2-F3. The product is marketed under the name Rezdiffra and is a selective THR-β agonist. Phase III clinical data showed that 50% of patients achieved NASH resolution or fibrosis improvement, more than 70% of patients had an MRI-PDFF decrease of more than 30%, and over 80% of patients achieved fibrosis reversal or no progression. As the first approved NASH drug, Resmetirom marks a milestone in NASH drug development. 🎉 Secondly, #IonisPharmaceuticals announced positive results from their Phase II clinical trial of their antisense oligonucleotide (ASO) therapy #ION224 for the treatment of metabolic dysfunction-associated fatty liver disease (MASH). The analysis showed that ION224 achieved the primary endpoint of histological improvement in liver tissue at both doses (120 mg and 90 mg), as well as achieving the important secondary endpoint of MASH regression. These breakthroughs represent significant progress in the development of treatments for NASH/MASH and provide hope for patients suffering from these conditions. #NASH #MASH #Resmetirom #MadrigalPharmaceuticals #ION224 #IonisPharmaceuticals

We sadly, have another example highlighting concerns with the accelerated approval pathway. Oxbryta first received accelerated approval in 2019 for sickle cell disease age 12 and older and then received additional accelerated approval in 2021 for age 4 and up. Pfizer, the manufacture, is pulling it from all markets and stopping all trials involving the agent as of last week. Why? "Pfizer’s decision came a day before European regulators met to review Oxbryta’ safety. The European Medicines Agency started its review because of data from two trials, one that had a higher-than-anticipated number of deaths and another that showed more deaths among patients taking Oxbryta compared with those on placebo." Sickle Cell Disease is an indication where we need more treatment options, more focus, and more funding. The accelerated approval pathway was designed to bring these treatments forward quicker. But how do we reconcile approvals too soon leading to harm when additional studies and full approval may have caught this? #Pharmacist #Pharmacybenefits #Sicklecelldisease #accerlatedpathway https://lnkd.in/gDGxcGBs