Sage Therapeutics

2024 saw highs and lows in neuroscience drug development, including FDA approval of BMS's Cobenfy for schizophrenia and Eli Lilly's donanemab for Alzheimer's, alongside failures like Sage Therapeutics' dalzanemdor in Parkinson's, Alzheimer's, and Huntington's. Despite setbacks, progress continues in treating intractable neurological diseases.

Vagus nerve stimulation therapy showed significant improvement in depressive symptoms, quality of life, and daily functioning for patients with treatment-resistant depression, according to a national clinical trial led by WashU Medicine. The study involved nearly 500 participants, with activated devices showing more time with improved symptoms and better quality of life. The therapy, if covered by CMS, could become more accessible.

Sage acquires Mary Ann Liebert, Inc., a media company publishing over 100 peer-reviewed journals in biotechnology, life sciences, clinical medicine, public health, policy, and technology. Journals will continue under the Mary Ann Liebert, Inc. name, with Sage publishing a segment of its life sciences and medical journals under the same imprint. Both companies are mission-driven and founded by pioneering women in academic publishing.

2024 saw significant clinical successes and failures in biopharma, with Vertex, BMS, and Alnylam achieving notable wins, while AbbVie, Sage Therapeutics, Pfizer, and Gilead faced setbacks in neurological and other indications. Despite high failure rates in drug trials, particularly in CNS diseases, some companies advanced new treatments, while others faced challenges in their pipelines.

Dr. Horan, Dr. Sauder, Dr. Ramsay, and Dr. Yohn are Bristol Myers Squibb employees. Dr. Harvey has affiliations with multiple companies, including Bristol Myers Squibb. Dr. Ramsay holds Bristol Myers Squibb stock. Dr. Keefe consults for various companies and has received royalties. Dr. Davis consults for several companies. Dr. Paul has roles at Karuna Therapeutics, Sage Therapeutics, and Voyager Therapeutics. Dr. Brannan was with Karuna Therapeutics and now consults for Bristol Myers Squibb.

UniQure aligned with FDA on accelerated approval pathway for Huntington’s disease gene therapy AMT-130, using Phase I/II studies and composite Unified Huntington’s Disease Rating Scale as primary basis for Biologics License Application (BLA).

Novartis pays $1 billion upfront to license PTC518, a Huntington's disease drug, with potential for $1.9 billion in milestone payments and royalties. PTC retains 40% of U.S. profits and leads Phase 2 completion and FDA discussions. The deal is expected to close in Q1 2025.

Novartis to pay up to $3 billion for PTC Therapeutics' experimental Huntington's disease drug, PTC518, in a deal that includes $1 billion upfront and potential milestone payments of $1.9 billion. The agreement reflects Novartis' commitment to neurodegenerative disease research and PTC's focus on RNA splicing therapies.

New treatments like COMP360, Zuranolone, LY03005, REL-1017, Seltorexant, and ABV-1504 are expected to significantly transform the major depressive disorder (MDD) market. MDD affects 44 million diagnosed cases in the 7MM, with moderate cases being the most prevalent. Treatment approaches include pharmacological interventions, psychotherapy, and lifestyle changes. The market size for MDD is projected to grow from USD 7.1 billion in 2023, driven by disease awareness and new product launches.

CVS Caremark adjusts diabetes drug coverage for 2025, removing older therapies like V-go Infusion Pump and Merck's Janumet/Janumet XR, in favor of newer products and generics. Notable additions include Twiist infusion system, Zituvimet, and biosimilars to Lantus. CVS also adds HIV, cancer, and mental health treatments to its formulary.