In one of the most highly anticipated clinical trial readouts this year, an #RNA silencing treatment developed by Alnylam Pharmaceuticals reduced the risk of death, heart-related hospital visits and hospitalizations by 28% compared to placebo in patients with a heart muscle disease.
The results, announced Monday, pave the way for Alnylam’s therapy vutrisiran to become the first RNA silencing therapy on the market for ATTR amyloidosis with cardiomyopathy, a condition in which misfolded proteins build up in the heart and can result in heart failure. If approved, Alnylam could enter a multibillion-dollar market currently dominated by Pfizer.
#clinicaltrials#clinicalresults#readout#RNAsilencing#heartmuscledisease#vutrisiran#heartdisease#cardiology#cardio#ATTRcardiomyopathy#patients#PhaseIII#clinicaldata
Cardiol Therapeutics Inc. (@cardiolrx) recently announced positive topline results from its Phase II MAvERIC-Pilot study, investigating the impact of CardiolRx™ on patients with symptomatic recurrent pericarditis. The study enrolled 27 adult patients across prominent clinical sites in the U.S., including the Mayo Clinic, Cleveland Clinic, and Massachusetts General Hospital.
The results demonstrated a significant reduction in pericarditis pain, with the mean pain score dropping from 5.8 at baseline to 2.1 (64%) after eight weeks of treatment. Additionally, 80% of patients with elevated baseline C-reactive protein (CRP) levels achieved normalization, indicating a substantial reduction in inflammation.
'We are delighted to share the exceptional primary endpoint data from the MAvERIC-Pilot study, which demonstrated that oral administration of our small molecule CardiolRx™ led to marked reductions in pericarditis pain and inflammation,' said David Elsley, President & CEO of Cardiol Therapeutics.
With an estimated 38,000 patients in the U.S. experiencing recurrent pericarditis annually, the need for effective treatments is critical. Cardiol Therapeutics aims to provide an accessible and non-immunosuppressive therapeutic option, potentially surpassing existing therapies. The positive results set the stage for a forthcoming Phase III trial.
#RecurrentPericarditis#CardiolTherapeutics#CardiolRx#ClinicalTrials#Biotechnology#Healthcare
@cardiolrx @mayoclinic @clevelandclinic @massgeneralnews
New, Emerging Treatment Options for Patients With Multiple Myeloma: In addition to emerging treatment options, the speakers addressed the importance of individualized decision-making when treating patients with multiple myeloma. #finance#pharmacy#lifesciences
From Research to Practice: New Treatment Modalities for Patients With Multiple Myeloma: Ryan Haumschild discusses the evolving treatment landscape for patients with multiple myeloma. #finance#pharmacy#lifesciences
Another day, another life-changing scientific break through.
Parkinson's particularly hits a raw nerve. Anyone who's seen a senior citizen suffering from this terrible affliction has got to feel some joy today.
The bizarre paradox of America:
The world's best medical innovation ecosystem coupled with the world's worst care delivery system.
And "system" is being generous.
The better word is probably "situation."
#Biotech#Biotechnology#Bigpharma#Pharmaceutical#Parkinsons#Neurology
Metabolic Disorders Solve Programme
We see that metabolic disorders are increasing day by day in the world and many people and organizations are trying to solve them. They make everyday new medicine combinations. In this way, all Allopathic, Ayurveda, Homeopathic, colour path, Iso path, naturopathy, Antipathy, Zyso pathy, Bio-chemic- path, fruit path, vitamin path, home path, acupressure path, acupuncture path, spiritual path, rake path electro-homoeopath, leaf path, flower path, Unani, Siddha, and Every country have Indigenous systems of medicine, Traditional systems of medicine but all are failed to cure diabetes and metabolic disorders
All kinds of metabolic disorders first time has no signs and symptoms and no discomfort, but when problems arise in the body we go to the Doctor because they develop very slowly and produce many kinds of diseases. We see that all kinds of metabolic disorders take place due to mutations of enzymes, lipids, and any food we eat which means we are not eating properly as required for digestion and mixing all enzymes. To solve this problem I wrote a Book (English) “Your Health Is In Your Mouth” (Hindi) “Aap Ka Sawasth Aap Ke Muh Me” You can find in a Google search
Congratulations to Daiichi Sankyo on its Enhertu's new key indications approved by FDA! The pioneering new-gen ADC drug by Daiichi Sankyo US was just accelerated approved by FDA for adult patients with unresectable or metastatic HER2-positive (IHC3+) solid tumors who have received prior systemic treatment and have no satisfactory alternative treatment options. Enhertu is now the first and only tumour-agnostic approval of a HER2-directed therapy and ADC by FDA.
ADC is one of topics of Chinese Antibody Society's 2024 Annual Conference which will take place in Cambridge, MA, USA, on Saturday, May 11, 2024. You are welcome to register for the conference right NOW via the following link.
https://lnkd.in/ecmFPA4v#antibodies#antibody#antibodytherapeutics#mabs#mab#antibodydiscovery#adc#adcs#antibodydrugconjugate # #antibodydrugconjugates#annualmeeting#annualconference
FDA - La Administración de Alimentos y Medicamentos 𝐆𝐑𝐀𝐍𝐓𝐒 𝐀𝐂𝐂𝐄𝐋𝐄𝐑𝐀𝐓𝐄𝐃 𝐀𝐏𝐏𝐑𝐎𝐕𝐀𝐋 𝐓𝐎 𝐋𝐈𝐒𝐎𝐂𝐀𝐁𝐓𝐀𝐆𝐄𝐍𝐄 𝐌𝐀𝐑𝐀𝐋𝐄𝐔𝐂𝐄𝐋 𝐅𝐎𝐑 𝐅𝐎𝐋𝐋𝐈𝐂𝐔𝐋𝐀𝐑 𝐋𝐘𝐌𝐏𝐇𝐎𝐌𝐀
Approval Date: May 15, 2024
🔵 Drug: Lisocabtagene maraleucel (Breyanzi, Juno Therapeutics, Inc. and Bristol Myers Squibb)
🔵 Indication: For adults with relapsed or refractory follicular lymphoma (FL) after two or more prior lines of systemic therapy.
🔵 Study: TRANSCEND-FL (NCT04245839)
🔵 Design: Phase 2, open-label, multicenter, single-arm trial
🔵 Participants: Adults with relapsed/refractory FL after ≥2 lines of systemic therapy (including an anti-CD20 antibody and an alkylating agent).
🟢 Primary Efficacy Population: 94 patients with PET-positive disease at baseline or after bridging therapy, received conforming product within intended dose range, and had ≥9 months follow-up from first response.
🟢 Overall Response Rate (ORR): 95.7% (95% CI: 89.5, 98.8)
🟢 Duration of Response (DOR): Median not reached (NR) after median follow-up of 16.8 months (95% CI: 16.3, 17.0)
🟡 Common Adverse Reactions (≥20%):
🔸 Cytokine release syndrome (CRS)
🔸 Headache
🔸Musculoskeletal pain
🔸Fatigue
🔸Constipation
🔸Fever
Alongside tisagenlecleucel (Kymriah Novartis) and axicabtagene ciloleucel (Yescarta Kite Pharma), it becomes the third CAR-T therapy approved for Follicular Lymphoma by FDA.
#cartcell#fda#hematology#clinicaltrial
Congratulations to Daiichi Sankyo on its Enhertu's new key indications approved by FDA! The pioneering new-gen ADC drug by Daiichi Sankyo US was just accelerated approved by FDA for adult patients with unresectable or metastatic HER2-positive (IHC3+) solid tumors who have received prior systemic treatment and have no satisfactory alternative treatment options. Enhertu is now the first and only tumour-agnostic approval of a HER2-directed therapy and ADC by FDA.
ADC is one of topics of Chinese Antibody Society's 2024 Annual Conference which will take place in Cambridge, MA, USA, on Saturday, May 11, 2024. You are welcome to register for the conference right NOW via the following link.
https://lnkd.in/ecmFPA4v#antibodies#antibody#antibodytherapeutics#mabs#mab#antibodydiscovery#adc#adcs#antibodydrugconjugate # #antibodydrugconjugates#annualmeeting#annualconference
CAR T Cell, Bispecific Therapies: Enhancing Treatment for Patients With Hematological Malignancies: Zahra Mahmoudjafari, PharmD, MBA, BCOP, FHOPA, discusses the impact of CAR T cell and bispecific therapies in treatment of patients with hematological malignancies. #finance#pharmacy#lifesciences
The European Medicines Agency (EMA)'s Committee for Medicinal Products for Human Use (CHMP) has recommended the expansion of CAR-T #celltherapy Carvykti (developed by Johnson & Johnson and Legend Biotech) for earlier treatment in patients with multiple myeloma.
The proposed change would see Carvykti move to a second-line treatment, opening the therapy up to all patients who have undergone at least one line of therapy.
Currently only approved for treatment of patients who have been through three or more lines of treatment, the expansion would represent a key step forward for the cell therapy industry and would see the lifesaving therapy become accessible to tens of thousands of patients every year.
Read more in BioSpace: https://bit.ly/4c00kYP