ChromaDex: Promising Study of Nicotinamide Riboside (NR) in Heart Failure With Reduced Ejection Fraction

 This is the first study to investigate the safety and tolerability of NR in a randomized, placebo-controlled trial of patients with heart failure, marking a milestone for future clinical research

ChromaDex Corp. (NASDAQ:CDXC) today announced promising findings from a clinical study, as reported in the peer-reviewed journal Journal of the American College of Cardiology (JACC): Basic to Translational Science by a team of scientists led by Dr. Kevin O’Brien, Division of Cardiology, Department of Medicine, in collaboration with Dr. Rong Tian, Mitochondria and Metabolism Center, Department of Anesthesiology and Pain Medicine, University of Washington School of Medicine, Seattle, Washington, USA. The clinical study was part of the ChromaDex External Research Program (CERP™) and investigated the safety and tolerability of the company’s proprietary Niagen® ingredient, patented nicotinamide riboside or NR, in Stage C heart failure patients with reduced ejection fraction (HFrEF), which occurs when the left ventricular ejection fraction (LVEF) is 40% or less. Additionally, the effects of NR on white blood cells’ mitochondrial respiratory function, inflammation and whole blood nicotinamide adenine dinucleotide (NAD+) levels were assessed. The promising results from this study demonstrate that high-dose NR was safe and well-tolerated, almost doubling whole blood NAD+ levels, increasing white blood cell mitochondrial respiratory function and decreasing the expression of inflammatory markers. This study marks a major milestone as it is the first study to investigate the safety and tolerability of NR in a randomized, placebo-controlled trial of patients with heart failure, a crucial step that will pave the way for future clinical research.

https://www.biospace.com/article/releases/chromadex-shares-promising-findings-from-clinical-study-showcasing-the-safety-and-tolerability-of-nicotinamide-riboside-nr-in-heart-failure-with-reduced-ejection-fraction/

Verastem Update: 1 FDA Meet on Horizon, 1 Trial Miss

 FDA Meeting Planned for Q4 to Discuss Regulatory Path Forward Based on Encouraging Results to Date in Ongoing RAMP 201 Trial in LGSOC

Results of Part A of RAMP 202 Trial in KRAS G12V-Mutant NSCLC Show VS-6766 ± Defactinib Did Not Meet Criteria to Continue to Expansion Phase

RAMP Trials with VS-6766 Combinations in KRAS G12C-Mutant NSCLC and Frontline Metastatic Pancreatic Cancer on Track

Newly Issued Patents Extend Coverage of VS-6766 and VS-6766 + Defactinib to 2038 and 2040

https://finance.yahoo.com/news/verastem-oncology-announces-ramp-vs-110000584.html

Positive Data at AAO Show Utility, Versatility of Clearside Suprachoroidal Space Platform

  Favorable Safety Profiles and Encouraging Efficacy Data Reported in Clinical Trials in the Suprachoroidal Space (SCS^®) Across Multiple Therapeutic Areas using Clearside’s SCS Microinjector^® -

- SCS Microinjector^® Featured in Multiple Oral and Poster Presentations at Recent American Academy of Ophthalmology (AAO) 2022 Annual Meeting -

Clearside Biomedical, Inc. (NASDAQ:CLSD), a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS^®), announced today that clinical data from multiple internal and partnered programs were presented at the recent 2022 Annual Meeting of the American Academy of Ophthalmology (AAO), the world's largest association of eye physicians and surgeons.

https://www.biospace.com/article/releases/positive-data-presentations-at-aao-annual-meeting-demonstrate-utility-and-versatility-of-clearside-biomedical-s-proprietary-suprachoroidal-space-platform/

Takeda Pharmaceutical to Discontinue Manufacturing of Natpar/Natpara by End of 2024

 Hypothyroidism treatment

https://www.marketscreener.com/quote/stock/TAKEDA-PHARMACEUTICAL-COM-6491073/news/Takeda-Pharmaceutical-to-Discontinue-Manufacturing-of-Natpar-Natpara-by-End-of-2024-41922382/

Sanofi wagering $400M in biobucks to go after muscular dystrophy with RNA

 Sanofi has joined the pile-on of drug developers targeting one of the biggest unmet needs in muscular dystrophy, committing to $30 million in upfront and near-term payments for global rights to miRecule’s preclinical prospect.

The deal will give Sanofi control of an anti-DUX4 RNA therapy that is in lead development as a treatment for facioscapulohumeral muscular dystrophy (FSHD). Sanofi plans to combine the therapy with its muscle-targeted nanobody technology to create an antibody-RNA conjugate (ARC). ARCs are designed to enable the targeted delivery of RNA. 

Multiple technologies underpin the plan. Sanofi is relying on miRecule for the anti-DUX4 RNA therapy and the conjugation and formulation chemistry to join it to the nanobody. The Big Pharma is supplying its nanobody, part of a class of therapeutic proteins based on single-domain antibodies that it began developing through its takeover of Ablynx.

By bringing together the technologies, Sanofi is trying to create a best-in-class therapy that selectively targets and suppresses the underlying cause of FSHD in muscle tissue. If the program is a success, the therapy could enable patients to resume their normal course of aging free from the effects of FSHD.

DUX4 has attracted multiple drug developers: Fulcrum Therapeutics is running a phase 3 trial of an oral small molecule designed to reduce expression of the gene; Arrowhead Pharmaceuticals has an RNAi prospect in preclinical development; and Epic Bio is seeking to suppress DUX4 expression using its gene expression modulation system.

Sanofi is betting $30 million in a mix of an upfront fee and near-term milestones to join the race. As the therapy advances, miRecule is in line to receive development, regulatory and commercial milestone payments that top out just short of $400 million, plus tiered royalties on global net sales. 

https://www.fiercebiotech.com/biotech/sanofi-spies-mirecule-therapy-wagering-400m-biobucks-go-after-muscular-dystrophy-rna

Biohaven Sets New Course with $258 Million in Cash, Proven Team, Deep Pipeline

 

• Broad therapeutic research and development portfolio includes more than 13 clinical and pre-clinical programs with a focus on neuroscience and rare disorders including epilepsy, pain and mood disorders, obsessive compulsive disorder (OCD), spinocerebellar ataxia (SCA) and spinal muscular atrophy (SMA).

• Excitement mounting for clinical stage neuroscience program in Kv7 Ion Channel Modulation which targets key subunits involved in neuronal signaling and plays a critical role in regulating the hyperexcitable state in epilepsy and potentially other central nervous system (CNS) disorders. 

• Biohaven retains Board and key management team with established legacy of bringing the market-leading medicine Nurtec® ODT (rimegepant) to patients; Names Bruce Car, Ph.D. as Chief Scientific Officer; Irfan Qureshi, M.D. as Chief Medical Officer; and Tanya Fischer, M.D., Ph.D. as Chief Development Officer and Head of Translational Medicine.

Biohaven Ltd. (NYSE: BHVN) launched today as a new publicly traded company focused on delivering innovative life-changing treatments for neurological and neuropsychiatric diseases, including rare disorders, leveraging its proven drug development capabilities and proprietary technology platforms to advance a pipeline of best-in-class therapies. As of today, Biohaven has officially begun operating as a separate independent entity as part of the acquisition agreement with Pfizer in May 2022. The company, led by Vlad Coric, M.D. as Chairman and Chief Executive Officer, launched with approximately $257.8 million in cash at the distribution and no debt.

https://finance.yahoo.com/news/biohaven-sets-course-258-million-113000401.html

Inhibrx's Experimental Drug For Inherited Disease Capable For Accelerated FDA Approval

 

• Based on discussions with the FDA, Inhibrx Inc INBX has the potential to pursue accelerated approval for INBRX-101 for emphysema due to alpha-1 antitrypsin deficiency (AATD) using functional alpha-1 antitrypsin (AAT) serum levels as the surrogate endpoint. 
• Inhibrx also detected INBRX-101 in the bronchoalveolar lavage fluid (BALF) samples from all AATD patients tested in the Phase 1 study.
• Inhibrx plans to initiate in Q1 of 2023 a potential registration-enabling trial using functional AAT as a surrogate endpoint.
• Based on data from the completed Phase 1 study, INBRX-101 is predicted to maintain patients above the lower threshold of the normal range and achieve an average level of functional AAT.
• The FDA also requested additional data on the correlation between functional AAT levels and the clinical benefit of AATD to further support serum AAT levels as a surrogate endpoint that is reasonably likely to predict clinical benefit.

https://www.benzinga.com/general/biotech/22/10/29136708/inhibrxs-experimental-drug-for-inherited-disease-capable-for-accelerated-fda-approval