Alphalyse’s Post

An initiative set up by the FDA to accelerate the development of therapies for rare disease in the same way that Operation Warp Speed delivered COVID-19 vaccine has chosen its first pilot programme candidates. The Support for Clinical Trials Advancing Rare Disease Therapeutics (START) scheme was set up last year to address challenges associated with rare disease drug development and speed up the regulatory process. Denali Therapeutics, Neurogene, Larimar Therapeutics, and Grace Science will be the first companies to benefit from the FDA's higher levels of guidance and advice in areas like clinical trial design, #patient population selection, use of nonclinical information, and #product characterisation. The #pilot is expected to eventually include #six or more projects. #FDA #Neurogene #Denalitherapeutics #Larimartherapeutics #Gracescience #Rarediseases #Drugdevelopment This pilot will set the stage for many more cases in #regulatory affairs and #accelerated drug development to benefit #patients in the U.S. and #worldwide.

⏭ Here are some recent biopharma news stories ¹ ² ³ ⁴: Ø Bristol Myers’ leukemia drug Sprycel faces competition from generic versions in the US market Ø The US FDA rejected Novo Nordisk’s application for a new insulin product Ø The US Federal Trade Commission is suing CVS Caremark, Express Scripts, and Optum Rx over their drug price practices Ø AbbVie’s Humira lost more market share to biosimilars after being removed from CVS Caremark’s formularies Ø Pfizer and Evotec are collaborating on early discovery research for metabolic and infectious disease therapeutics Ø Roche recalled Susvimo’s ocular implant, insertion tool, and initial fill kit due to unsatisfactory test results Ø Novo Nordisk is investing $4.1 billion in a new fill/finish manufacturing facility in North Carolina. Source:1 https://lnkd.in/g7XdPHEf 2 https://lnkd.in/gQc9nbYQ 3,4 https://lnkd.in/gZYKFhs6 https://lnkd.in/gDgEyQ-t

🌟 The Biotech News You Missed This Week! 🌟 💊 Eli Lilly and Company Acquires Morphic Holding LLC Lilly will pay $3.2B for the Waltham-based biotech developing treatments for inflammatory bowel disease. 🧬 IDEAYA Biosciences’ Promising Results A mid-stage study shows a 39% tumor response rate for IDE397 in bladder and lung cancer patients. 🛑 HilleVax’s Vaccine Trial Setback The norovirus vaccine failed in infants, prompting the company to halt development. 💰 AbbVie’s Massive Marketing Spend AbbVie leads with $145.7M spent on healthcare provider marketing in 2023, far surpassing its peers. 📉 GLP-1s and Cancer Risk Reduction GLP-1 drugs like Ozempic show significant reductions in risks for 10 obesity-related cancers compared to other diabetes medications. ⚖️ Medicare Drug Pricing Negotiation Update A judge ruled against Boehringer Ingelheim’s challenge, strengthening Medicare’s negotiation program. #Biotech #Healthcare #Pharma #CancerResearch #DrugDevelopment #Medicare #HealthcareMarketing

Did you know that this year, Rare Disease Day falls on the rarest date of the year - 29 February? A 'rare' day for 'rare' diseases! This leap year, let's use this unique alignment to leap towards progress. Let's unite, amplify voices, and raise awareness for the millions affected by rare diseases. Here's to a weekend of thought, and we are looking forward to a momentous #RareDiseaseDay! Everything; For Our Rare Patients Struggling with Rare Diseases... A hope for rare patients to access treatment; we will continue to collaborate with our physicians and developer pharmaceutical companies to increase treatment options and improve the success of their treatments. You only need to take one step to work with us Fatih AKTAŞ BiotchPharma Pharmaceutical Marketing and Consulting Company Biotech CRO Medical Pharmaceutical Research Consultancy Trade Limited #RareDiseaseDay! #ClinicalTrial #RareDiseases #OrphanDrugs #ExpandedAccessPrograms #NamedPatientPrograms #EarlyAccessPrograms #CompassionateUse #BusinessConsultant #SalesMarketing #ProjectManagement #Hematology #Oncology #GeneticDiseases #MetabolicDiseases #Cardiology

🚨 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

📰💊 Here is our weekly round-up of the top 3 Pharma breakthrough stories you need to know 💊📰 ✅ Protein Degraders: Pioneering New Therapies for Cancer and Neurological Diseases: Protein degraders are emerging as a breakthrough drug class, targeting disease-causing proteins to treat cancers and neurological conditions. Arvinas Therapeutics' vepdegestrant, a promising breast cancer therapy, could be the first approved drug in this space by 2025, opening doors for further advancements in the field. We commend Arvinas CEO, John Houston & his team Craig Crews & Ray Deshaies, Ph.D. https://lnkd.in/d7BH2xK5 ✅ On-Dose Authentication: A New Era in Combating Pharmaceutical Counterfeiting: Pharmaceutical companies are combating counterfeiting by implementing on-dose authentication, including methods like SECURtracers, which integrate micro-engraved tracers into drug coatings. This innovative approach enhances drug security by allowing direct product verification, complementing existing packaging-based measures, and ensuring authenticity throughout the supply chain. https://lnkd.in/dg6hYC4Q ✅ GLP-1 Obesity Drugs Show Low Suicide Risk, But Experts Call for Continued Monitoring: New research suggests GLP-1 obesity drugs like Wegovy do not significantly raise the risk of suicide or mental health issues, according to studies from Sweden, Denmark, and Novo Nordisk trials. However, experts recommend continued monitoring, especially for patients with preexisting mental health conditions, as more data is needed to fully understand long-term effects. https://lnkd.in/d5XmUpXE #NeedToKnow #PharmaBreakthrough #PharmaNews #PharmaUpdates #HealthcareAdvances #DrugDevelopment 

Landscape of toxin-neutralizing therapeutics for snakebite envenoming 🐍   Our co-founder and CSO, Cecilie Knudsen, has co-authored this paper with Juliette Borri from Policy Cures Research and others, which has just been published in PLOS Neglected Tropical Diseases.   The paper explores the current landscape of snakebite envenoming therapeutics (both those already marketed and those in R&D), to develop a proper overview of treatments available (or soon to be) for snakebite victims globally.   At VenomAid, we work on developing snakebite diagnostics that reflect the available therapeutics and facilitate early and correct treatment, which is why we follow the development of toxin-neutralising therapeutics closely. One the most interesting new R&D trends described in this paper, is the increased focus on therapeutics based on recombinant antibodies and small molecule therapeutics, which target toxin-(sub)-families rather than whole venoms. This trend could have implications for snakebite diagnostics, where one of the big questions is which antigens are most crucial to detect.    You can read the full article for free below 🔬 #snakebite #antivenom #diagnostics

US Food and Drug Administration (FDA) has chosen Moderna's investigational therapeutic, mRNA-3705, for the prestigious Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program!   mRNA-3705 is a potential game-changer for individuals battling methylmalonic acidemia (MMA) due to methylmalonic-CoA mutase (MUT) deficiency. This selection underscores Moderna's commitment to pushing the boundaries of mRNA technology beyond vaccines, aiming to address critical unmet medical needs.   MMA is a rare, life-threatening disorder with limited treatment options, making this milestone even more significant. mRNA-3705, encapsulated within Moderna's proprietary lipid nanoparticle (LNP), aims to restore missing or dysfunctional proteins that cause MMA, potentially revolutionizing treatment for this debilitating condition.  #Moderna #MedicalInnovation #mRNA3705 #RareDiseaseTherapeutics #STARTProgram #ShantiPharmaceuticals

Did you know that this year, Rare Disease Day falls on the rarest date of the year - 29 February? A 'rare' day for 'rare' diseases! This leap year, let's use this unique alignment to leap towards progress. Let's unite, amplify voices, and raise awareness for the millions affected by rare diseases. Here's to a weekend of thought, and we are looking forward to a momentous #RareDiseaseDay! Everything; For Our Rare Patients Struggling with Rare Diseases... A hope for rare patients to access treatment; we will continue to collaborate with our physicians and developer pharmaceutical companies to increase treatment options and improve the success of their treatments. You only need to take one step to work with us Fatih AKTAŞ BiotchPharma Pharmaceutical Marketing and Consulting Company Biotech CRO Medical Pharmaceutical Research Consultancy Trade Limited #RareDiseaseDay! #ClinicalTrial #RareDiseases #OrphanDrugs #ExpandedAccessPrograms #NamedPatientPrograms #EarlyAccessPrograms #CompassionateUse #BusinessConsultant #SalesMarketing #ProjectManagement #Hematology #Oncology #GeneticDiseases #MetabolicDiseases #Cardiology

We are living in exciting times, with potential therapies for PCD beginning to be developed by pharma, including Ethris. “Beyond vaccines, we’re now looking toward mRNA’s use in therapeutics and opening additional routes of delivery beyond intravenous administration, including nebulization and nasal sprays for inhaled delivery,” reports Carsten Rudolph, PhD, the CEO of Ethris. “In the near future, inhaled mRNA therapies have the potential to treat chronic or genetic diseases of the lung, such as chronic obstructive pulmonary disease, primary ciliary dyskinesia, and pulmonary alveolar proteinosis.” 📲 Read the full article here: https://lnkd.in/eETjWETv