CHEManager International’s Post

Yesterday was #RareDiseaseDay. The annual observance highlights how far the #biopharma industry has come and serves as a wake-up call for how far it must still go to meet the needs of #raredisease patients. Despite recent investment and breakthroughs for #rarediseases, experts told BioSpace that helping a much larger patient population will require cross-sector support and desperately needed funding. We looked at some of the investments to date, as well as the biopharma industry’s promising pipelines and what is required to bring more groundbreaking therapies to patients with unmet medical needs. #Patientadvocacy groups have been critical to the development of the first marketed drugs for certain rare diseases, including progeria and Friedreich's ataxia. We spoke with leaders of these groups formed by families coping with rare diseases, providing case studies on their partnerships with biopharma companies. https://lnkd.in/g7R6hU23 #RareDiseaseDay2024 #genetherapy #genetherapies #duchenne #duchennemusculardystrophy #biopharmaceuticals

Advancements in Lupus Nephritis Treatment: A Promising Outlook for Patients Lupus nephritis, a severe manifestation of systemic lupus erythematosus (SLE), presents a complex challenge in the realm of autoimmune diseases. However, recent advancements in understanding its pathogenesis and targeted therapies have significantly improved outcomes for affected individuals. With a growing emphasis on precision medicine, researchers are unraveling the intricate molecular mechanisms underlying lupus nephritis, paving the way for novel therapeutic interventions. From biologics targeting specific immune pathways to innovative immunomodulatory agents, the landscape of lupus nephritis treatment is witnessing a paradigm shift towards tailored approaches that aim to mitigate disease progression and preserve renal function. Know more info:https://lnkd.in/d-hxcEf4 Moreover, the emergence of biomarkers for disease activity and response to treatment has revolutionized patient management, enabling clinicians to make informed decisions and optimize therapeutic strategies. In parallel, ongoing clinical trials evaluating novel drug candidates offer hope for further improvements in efficacy and safety profiles. As collaborative efforts between academia, pharmaceutical companies, and regulatory agencies continue to accelerate research and development in this field, the future holds promise for individuals battling lupus nephritis. By harnessing the power of precision medicine and leveraging innovative therapeutic modalities, the journey towards better outcomes and enhanced quality of life for patients with lupus nephritis is within reach. #LupusNephritis #AutoimmuneDiseases #PrecisionMedicine #Immunotherapy #Biomarkers #ClinicalTrials #TherapeuticInnovations

As we kick off Rare Disease Month this February, Gilead Sciences' recent acquisition of CymaBay Therapeutics for $4.3 billion is a thrilling announcement. The journey from setbacks in 2019 to a potential best-in-class drug for Primary Biliary Cholangitis (PBC) is a testament to the resilience and innovation in the field. CymaBay's dedicated research in liver disease, resulting in seladelpar's significant improvements in biomarkers and symptom management for PBC, aligns perfectly with Gilead's mission to expand their liver disease portfolio. This acquisition not only marks a pivotal moment for Gilead but also offers new hope to PBC patients. It highlights the importance of persistence in the face of challenges and the impact of dedicated research in rare disease. Congratulations to both Gilead Sciences and CymaBay Therapeutics on this exciting milestone! #RareDiseaseMonth #GileadSciences #CymaBayTherapeutics #Innovation #Resilience #DedicatedResearch #LiverDisease #PrimaryBiliaryCholangitis #Seladelpar #competitiveintelligence

𝗚𝗶𝗹𝗲𝗮𝗱 𝘁𝗼 𝗕𝘂𝘆 𝗖𝘆𝗺𝗮𝗕𝗮𝘆, 𝗟𝗲𝗮𝗱 𝗟𝗶𝘃𝗲𝗿 𝗗𝗶𝘀𝗲𝗮𝘀𝗲 𝗔𝘀𝘀𝗲𝘁 𝗦𝗲𝗹𝗮𝗱𝗲𝗹𝗽𝗮𝗿 𝗳𝗼𝗿 $𝟰.𝟯𝗕 Gilead Sciences is acquiring CymaBay Therapeutics for $4.3 billion, focusing on seladelpar, a treatment for primary biliary cholangitis (PBC). The FDA has granted seladelpar priority review, with a decision expected by August 14, 2024. Seladelpar has shown promise in improving symptoms and disease markers in PBC patients. The acquisition, at $32.50 per share, aligns with Gilead's liver disease portfolio and is expected to be earnings neutral in 2025 if seladelpar is approved. This strategic move follows Gilead's recent efforts to bolster its presence in liver disease treatment and research. Key Highlights: Seladelpar, a selective peroxisome proliferator-activated receptor delta (PPARδ) agonist, demonstrated a 61.7% response rate in patients versus 20% in the placebo group for the primary composite endpoints in a Phase III trial. Seladelpar also showed a statistically significant improvement in pruritus among patients with moderate-to-severe itch over six months, maintained through 12 months. This acquisition follows Gilead's recent collaboration amendment with Arcus Biosciences, aimed at accelerating an anti-TIGIT program #metabolism #liverdisease #TIGIT #gilead

Chronic #HepatitisDelta is a devastating viral disease with no approved treatments in the United States. I'm energized to see today's preliminary results from Vir Biotechnology, Inc.'s Phase 2 SOLSTICE clinical trial, evaluating mono- and combination therapies in the treatment of #HepatitisDelta. It's days like today that make me even more excited to come to work tomorrow. #PatientsAreWaiting https://lnkd.in/gHHhgdVS

TG Therapeutics, Inc. gains WW rights to Precision BioSciences, Inc.'s allo CAR-Ts development programs across several autoimmune indications, and "expects to file an investigational new drug application for azercabtagene zapreleucel (azer-cel), a CD19-targeted CAR-T, around the middle of 2024. " Some interesting commentary coming out of the #JPM2024 on the potential of CAR-Ts in autoimmune conditions: "At the JP Morgan healthcare conference on Monday, FDA’s Peter Marks said the sentiment is strong for the CAR-T pivot into autoimmune diseases, despite questions lingering over a higher safety bar for less severe diseases that could stop the cell therapies in their tracks. “The risk/ benefit is still going to be favorable to move forward in those development plans,” he said, calling out settings like lupus nephritis and lupus cerebritis as potentially lethal diseases." https://lnkd.in/e84UER2z #TGTherapeutics #PrecisionBiosciences #alloCARTs #CARTs #CellTherapy #AutoImmune #Biotech #CorporateAffairs #Healthcare

Amylyx Pharmaceuticals has released interim results from its ongoing Phase II HELIOS trial, showing that its lead asset AMX0035 (sodium phenylbutyrate and taurursodiol) improved pancreatic function and glycemic control in adult patients with the rare neurodegenerative disease Wolfram syndrome. Previously available commercially in the U.S. under the brand name Relyvrio, AMX0035 won the FDA’s approval in September 2022 for the treatment of amyotrophic lateral sclerosis. However, in March 2024, Relyvrio failed the Phase III PHOENIX trial missing its primary endpoint of ALSFRS-R total score change at 48 weeks. Relyvrio also failed key secondary endpoints such as slow vital capacity and self-reported health status. Last week, Amylyx announced that it was voluntarily withdrawing Relyvrio from U.S. and Canadian markets. Patients currently on treatment will have the option to transition into a free program. #biotech #biopharma #Relyvrio #WolframSyndrome

Vertex Pharmaceuticals moves #inaxaplin into Phase III trials for APOL-1 mediated kidney disease (AKMD), targeting the disease at its genetic roots. Phase II data showed a significant reduction in proteinuria, spotlighting inaxaplin's potential to transform AKMD treatment. Key Takeaways - 🔹 Impact - Offers new hope to ~100,000 affected patients in the U.S. and Europe. 🔹 Insight - Early and persistent efficacy in reducing proteinuria, with a safety profile that supports its use. 🔹 Discussion Point - How could inaxaplin’s approval change the landscape of kidney disease treatment? #KidneyDisease #ClinicalTrials #HealthcareInnovation

Very interesting read, for those of you interested in extending both Healthspan and Lifespan.

Immutep Limited has entered into an agreement with a leading research institute, the Centre for Human Drug Research (CHDR), to perform a first-in-human clinical study of IMP761. IMP761 is a proprietary LAG-3 agonist antibody has been designed to restore balance to the immune system and address the underlying cause of many autoimmune diseases.  CHDR is a world-class institute in Leiden, the Netherlands specialising in cutting-edge early-stage clinical drug research. The trial is expected to begin mid-CY2024.  #LAG3 #immunotherapy #autoimmunedisease https://bit.ly/3Q60lRE