Biogen

Innovation in neuropsychiatry is reviving with approvals like Bristol Myers Squibb’s Cobenfy and Sage Therapeutics’ Zurzuave. Challenges include costly Phase III trials and lack of effective animal models for CNS disorders. Investment in neuroscience is cyclical, with unclear causality and biomarkers. Biogen’s Aduhelm and Eisai’s Leqembi highlight the complexities of launching treatments in neurodegenerative diseases. Early diagnosis and targeted patient selection are crucial, with AI and blood-based biomarkers offering potential solutions. Psychedelics show promise in neuropsychiatry, though regulatory and public perception hurdles remain. The field faces challenges due to the lack of biological biomarkers, but functional improvements and quality-of-life gains drive optimism and investment.

Alzheimer's researchers at the 2024 CTAD conference discussed the future of treatment, focusing on combination therapies, reducing ARIA risk, and novel approaches like gene therapy and stem cell treatment. Key topics included the potential of anti-amyloid antibodies in combination with other therapies, efforts to lower ARIA risk, and the presentation of promising data from gene therapy and stem cell studies.

New Huntington's disease therapies are nearing R&D finish lines, aiming to be the first disease-modifying treatments. Despite high-profile failures, companies like Wave, Prilenia, and uniQure are progressing towards regulatory approval. The field has gained momentum after decades of trial and error, with several mid-stage clinical trials underway. The ideal therapy would selectively lower mutant huntingtin and be widely distributed in the brain, though none currently exist.

The global spinal muscular atrophy market is projected to grow from USD 2,013.5 million in 2024 to USD 4,823.9 million by 2031, at a CAGR of 13.3%. Key drivers include FDA approvals for drugs like Zolgensma and Evrysdi, rising awareness, and increasing gene therapies. North America is expected to dominate due to its established healthcare infrastructure.

Eisai reduces Leqembi sales forecast to JPY 42.5 billion from JPY 56.5 billion due to sluggish uptake. The drug, partnered with Biogen, faces regulatory setbacks in EU and Australia and reimbursement issues. Eisai hopes new formulations and the AHEAD 3-45 trial results will boost sales.

The Patient Recruitment for Rare Disease Trials Summit tackles challenges like geographical dispersion and low awareness by exploring innovative strategies to efficiently identify and engage eligible participants, featuring actionable insights, knowledge on patient feedback integration, best practices for data sharing, and fair patient reimbursement discussions.

The Immunomodulators market is projected to grow from $248M in 2023 to $364.7M by 2030, driven by rising autoimmune diseases, advancements in biologics, and personalized medicine. Immunomodulators are crucial for treating immune-mediated diseases, with technological advancements expanding treatment options. The market segments include immunosuppressants, immunostimulants, and biologics, with biologics leading growth. Key regions like the U.S. and China show significant growth potential.

Ionis Pharmaceuticals (IONS) reported a narrower-than-expected Q3 2024 loss of 95 cents per share, with total revenues of $134 million, beating estimates. The company's shares have declined 22.9% YTD. Ionis receives royalties from Biogen for Spinraza and co-markets Qalsody with Biogen. Wainua, co-marketed with AstraZeneca, was approved in the U.S. and the UK, generating $30 million in milestone payments. Commercial revenues were $76 million, missing estimates. Ionis maintained its 2024 financial guidance, expecting total revenues over $575 million and an adjusted operating loss of less than $475 million.

In 2024, smaller deals have dominated pharma M&A, with no single deal surpassing $5 billion. Despite this, M&A remains crucial for refilling pipelines and supporting smaller biotechs. Notable deals include Vertex's $4.9 billion acquisition of Alpine Immune Sciences, Eli Lilly's $3.2 billion acquisition of Morphic, and Gilead's $4.3 billion purchase of CymaBay. Oncology and neuroscience have seen significant activity, with Novartis and AstraZeneca leading in oncology, and Bristol Myers Squibb and Lundbeck in neuroscience.

IMUNON reported Q3 2024 financial results, highlighting progress with IMNN-001 in advanced ovarian cancer and IMNN-101, a seasonal COVID-19 booster. IMNN-001 showed an 11.1-month OS improvement in OVATION 2 Study, with plans to start a Phase 3 study in Q1 2025. The company raised $10 million in a registered direct financing and added key leadership to support operational excellence.