Biogen

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.

Ionis Pharmaceuticals' shares rose 5% after FDA approved its RNA-targeted therapy, Tryngolza, for familial chylomicronemia syndrome (FCS), marking the first treatment for FCS in the U.S. The approval is supported by phase III BALANCE study results showing significant triglyceride-lowering and reduced acute pancreatitis attacks. Tryngolza is Ionis' first wholly-owned drug and independent commercial launch, expected by year-end. The company is also evaluating Tryngolza for severe hypertriglyceridemia and has collaborations with major drugmakers for various treatments.

Roche and Prothena's prasinezumab, targeting alpha-synuclein for Parkinson's, missed primary endpoint in phase 2b PADOVA trial but showed numerical delay in motor progression and positive trends on secondary endpoints. Roche plans further exploration and collaboration with Parkinson's community.

Biogen navigates Alzheimer's treatment Leqembi launch and pipeline shifts. Key growth drivers include Leqembi and Skyclarys for Friedreich's ataxia. Strategic moves like acquiring HI-Bio and financial stability with strong cash flow and gross margin support Biogen's prospects. Competitive landscape and regulatory challenges in Europe pose risks. Analysts' mixed outlook ranges from $175 to $294, emphasizing Leqembi's success and pipeline developments.

CMS requires Medicare Advantage plans to cover Qalsody (tofersen) for ALS patients with SOD1 mutations, reversing previous denials. The directive follows advocacy by the ALS Association, which emphasizes FDA-approved treatments should not be labeled as 'experimental'. Medicare Advantage plans must now stop classifying Qalsody as such and inform previously denied patients of new coverage policies.

The US biosimilar market surged in 2024, driven by FDA approvals and looming patent cliffs, with 63 biosimilars approved and 42 launched. Innovator companies like Novartis, Biogen, Pfizer, and Amgen are entering biosimilar development to mitigate sales erosion. Challenges include market access, reimbursement barriers, and HCP/patient perception. Education and transparency are crucial for biosimilar adoption, with AI tools potentially aiding HCPs.

Digital transformation in pharma is crucial, with 75% of organizations implementing Pharma 4.0. Key drivers include patient demands, regulatory changes, and competition. Top technologies include AI, IoT, blockchain, and digital twins. Strategic insights focus on R&D, supply chain, marketing, and patient engagement.

The FDA's accelerated approval pathway, enacted in 1992, has led to over 200 drugs reaching market based on surrogate endpoints. Despite recent withdrawals like Pfizer's Oxbryta and Takeda's Exkivity, the program has a strong track record, with over half of approvals converting to full approval. Challenges include failed confirmatory trials and the need for better biomarker understanding.

Molecular glue degraders, discovered serendipitously, have opened a new field of drug discovery, attracting Big Pharma investments. These small molecules enhance protein destruction by interacting with ligases, targeting 'undruggable' proteins. Major deals in 2024 include collaborations between Biogen, Novartis, Takeda, and Novo Nordisk, potentially worth over $6 billion. The therapeutic potential spans oncology, neurodegeneration, autoimmune, and cardiometabolic diseases. The discovery of cereblon as thalidomide's target in 2010 was pivotal, leading to further research and development in this space.

Focke Ziemssen, Michelle Sylvanowicz, Winfried M. Amoaku, Tariq Aslam, Bora Eldem, Robert P. Finger, Richard P. Gale, Laurent Kodjikian, Jean-François Korobelnik, Xiaofeng Lin, Anat Loewenstein, Paul Mitchell, Moira Murphy, David R. Owens, Nick Parker, Ian Pearce, Francisco J. Rodríguez, Jude Stern, S. James Talks, David T. Wong, Tien Yin Wong, and Jane Barratt disclose various financial interests including travel grants, personal fees, advisory board memberships, speaker fees, research sponsorship, and consulting roles with companies such as Allergan, AbbVie, Alimera, Bayer Healthcare, Biogen, Boehringer Ingelheim, Janssen, Novartis, Novo Nordisk, Merck, Regeneron, Roche, Sanofi, and others.