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🚨 Industry Insight: AN2 Therapeutics 🚨 AN2 Therapeutics, a California-based biotech firm, has recently hit a critical juncture in its quest to combat lung disease. Merely five months into the phase 3 segment of a pivotal trial for epetraborole, their leading clinical candidate aimed at treating mycobacterium avium complex (MAC) lung disease, the company has decided to press pause. Key Takeaways: 🛑 The pause comes after preliminary phase 2 results suggested the drug’s efficacy might be lower than anticipated. 🧪 Despite this setback, it’s important to note the decision wasn't triggered by safety concerns. 📊 AN2 Therapeutics remains optimistic, planning to release top-line data from the phase 2 trial this summer. Epetraborole is more than just a drug for AN2; it's a beacon of hope for patients with MAC lung disease—a condition notoriously tough to treat and with few effective options available. CEO Eric Easom emphasised the complexities of developing new treatments for such a challenging disease, reflecting the high stakes and high hopes resting on epetraborole’s success. As the biotech community watches closely, AN2 Therapeutics’ journey underscores the unpredictable nature of drug development, especially for diseases desperate for better treatment options. #AN2Therapeutics #Biotech #ClinicalTrials #LungHealth #PharmaNews

We're excited to share that our collaborative project on Fabry disease(LA-GLA, code name: HM14521/GC1134A) with Hanmi Pharmaceutical, has just earned Orphan Drug Designation(ODD) from the FDA! Living with Fabry disease means enduring the inconvenience of bi-weekly, hours-long IV treatments at the hospital. But here comes LA-GLA, a game-changer! It's the first-ever treatment to offer a once-a-month subcutaneous injection, breaking new ground beyond the limitations of existing therapies. LA-GLA is proving superior to current treatments, with significant improvements noted in kidney function, vascular health, and peripheral nerve disorders. This February, two pivotal studies showcasing its benefits were presented at the WORLD Symposium 2024 in San Diego. Buoyed by these promising results, we are gearing up to file an IND application this year. *Fabry disease is a genetic disorder linked to the X chromosome, caused by deficient levels of the enzyme alpha-galactosidase A. This deficiency leads to cellular toxicity and inflammation, progressively damaging various organs and potentially escalating to a severe, life-threatening condition. https://lnkd.in/evM9FCGT #Fabry #LAGLA #ODD #FDA #Innovation #Partnering #Collaboration

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

Huntington’s Disease Market Forecast 2034: Therapies, Clinical trials, prevalence, Treatment Algorithms by DelveInsight | MicuRx, Basilea pharmaceuticals, AbbVie, Cubist Pharms LLC, Nabriva Therapeuti: Huntington’s Disease Market As per DelveInsight, the Huntington’s Disease Market is anticipated to grow immensely in the coming years owing to the launch of upcoming therapies, research and development and the increase in the incident population of Huntington’s Disease in … Continue reading → #Business #Europe #HealthMedicine #PharmaceuticalsBiotech #US

A Drug Fails post Approval due to lack of Clinical Benefit and Concerns of Toxicity: Aduhelm abandoned For Alzheimer's Aducanumab (Aduhelm), a drug recently approved for the treatment of Alzheimer's Disease, was withdrawn from selling in the US. Biogen stopped an ongoing confirmatory clinical trial and abandoned its rights to the drug. Aducanumab (Aduhelm) was approved by the USFDA for the treatment of AD. The main questions about its approval was about lack of clear evidence of clinical benefit and concerns of toxicity. Learning: Only drug candidates which show meaningful clinical response with an acceptable safety profile (as opposed to objective responses whether CNS or oncology) in registration studies should be considered for full approval. Drugs granted accelerated approval (based on surrogate biomarkers or objective responses) should be evaluated for clinical benefit in confirmatory trials. https://lnkd.in/garPWSK8

Heading to the World Orphan Drug Congress in #Boston this week? On Wednesday, April 24 at 3:20 pm, Michael Fusakio and Samantha Z. will lead a fireside chat on "Rare but not Forgotten: Challenges and Opportunities for Rare Disease Drugs." You'll hear stories and lessons learned on the path to getting rare disease drugs on the market. To help further accelerate the development of novel drug and biological products for rare diseases, the #FDA launched START. This is a pilot program allowing for more communication with the agency to address development challenges. Read Mike Fusakio's review of the program and its benefits. https://hubs.la/Q02tRwPf0 Stop by the fireside chat to learn more about opportunities to advance your rare disease drug development. #WODC #OrphanDrugDesignation #RareDisease

Nearly 2,000 drug plants overdue for FDA checks after COVID delays, AP finds The firms that are overdue for safety and quality inspections represent about 42 per cent of the 4,700 plants that are currently registered to produce drugs for the US and previously underwent FDA review before May 2019, the AP found. The plants make hundreds of critical medicines, including antibiotics, blood thinners and cancer therapies. #drugsupplysafety I #drugplants I #qualityinspections I #drugcontaminationrisk I #genericdrugmakers I #pharmaceuticalindustry Read more: https://lnkd.in/guC8JKFG

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

The FDA recently published a draft guidance titled, “Optimizing the Dosage of Human Prescription Drugs and Biological Products for the Treatment of Oncologic Diseases,” to advance dosing protocols. They are currently evaluating public input before finalizing the policy. “With the emergence of targeted therapies and immunotherapies and improvement in the long-term prognosis for many cancers, we believe that oncology drug development needs to embrace new approaches that consider all available clinical and nonclinical data (not just short-term safety data) in real time.” Read more via the American Society for Clinical Pharmacology & Therapeutics: https://bit.ly/3PEVbw4 | #PrecisionMedicine #PrecisionDosing #OncologyDrugs #RealTimeData

An old one but a good one nonetheless. Successfully launching a rare disease drug in a patient-centric world requires a true commitment to understanding and engaging with the rare disease community in order to foster innovative methods for patient identification, strategically navigating patient access, and provide comprehensive support for patients and caregivers throughout their treatment journey. The core focuses of pharmaceutical companies haven't changed much, though I'd say that thanks to a rise in AI/ML, there has been an increase in the prominence and utilization of real-world data and evidence. Because the underlying drivers and pillars are still largely the same as they were a few years ago, we're likely going to see a complete shift in patient experience, driven by advancements in treatment accessibility, engagement, and real-world evidence generation, ultimately reshaping the landscape of rare disease therapeutics. #RareDiseases #DrugDevelopment #PatientCare #Healthcare