Piper Sandler has initiated coverage on ANI Pharmaceuticals (ANIP), a specialty pharmaceutical firm, with an ‘Overweight’ rating and a price target of $68. The firm highlights ANI’s successful transformation from a generics-focused company to a leader in rare disease therapies, driven by the success of its lead asset, Cortrophin Gel.
Italian pharmaceutical company Recordati has acquired the global rights to Enjaymo (sutimlimab), a treatment for cold agglutinin disease (CAD), from Sanofi for $825 million upfront and potential milestone payments of up to $250 million. The deal is expected to be immediately accretive to Recordati’s earnings and will be funded by existing cash and new debt facilities.
The orphan drugs market is experiencing significant growth, driven by advancements in biotechnology, increased understanding of rare diseases, and favorable regulatory environments. This market segment is projected to reach US$ 424.0 billion by 2034, driven by the increasing demand for personalized medicine solutions and the development of innovative treatments for rare diseases.
BioMarin Pharmaceutical, a leading rare disease specialist, remains optimistic about its future despite recent competition from Ascendis Pharma. Despite Ascendis Pharma’s positive Phase 3 trial results for achondroplasia, BioMarin believes its Voxzogo drug will continue to perform well. This confidence is based on Voxzogo’s broad label without age restrictions, providing a competitive edge. BioMarin is targeting a significant revenue increase and improved operating margins, driven by Voxzogo’s growth and a new business structure. The company is also exploring external business development opportunities to further diversify its portfolio.
Zevra Therapeutics’ Miplyffa (arimoclomol) has received FDA approval for treating neurological symptoms associated with Neimann-Pick disease type C (NPC) in adults and children. This is the first drug approved for this rare genetic disease, offering hope to patients suffering from its progressive neurological symptoms and organ dysfunction.
Stifel has initiated coverage on Inozyme Pharma (INZY), highlighting the potential of its lead candidate, INZ-701, an enzyme replacement therapy (ERT) for rare diseases. The analyst believes the drug has pipeline-in-a-product potential and forecasts a Buy rating with a price target of $16.
The Indian government has included primary hyperoxaluria, a life-threatening kidney, bladder, and urinary tract condition, under its National Policy for Rare Diseases, providing financial assistance of up to ₹ 50 lakh for patients requiring kidney transplants. This inclusion signifies the government’s commitment to supporting individuals battling rare diseases and ensuring access to life-saving treatments.
The global acute intermittent porphyria (AIP) market is projected to experience substantial growth, driven by advancements in AI, digital health technologies, and patient-centric drug development. AI-powered tools are enabling personalized treatment strategies and improved outcomes, while telemedicine and remote monitoring are revolutionizing AIP care delivery. This market analysis explores key trends, regional profiles, and prominent players shaping the future of AIP treatment.
Ascendis Pharma’s Yorvipath (palopegteriparatide) has received FDA approval for the treatment of hypoparathyroidism in adults. This approval fills a crucial gap in treatment options for this rare condition, particularly as Takeda’s Natpar/Natpara will be discontinued in 2024. Yorvipath is expected to be available in the first quarter of 2025.
Incyte (NASDAQ: INCY) has entered into an agreement to acquire Escient Pharmaceuticals, a privately held biotechnology company focused on developing novel therapeutics for immune and neuro-immune disorders. The transaction, valued at $750 million plus Escient’s net cash, is expected to close in the third quarter of 2024, subject to customary closing conditions. Upon completion, Incyte will gain exclusive rights to Escient’s portfolio of compounds, including EP262 and EP547, which target the MRGPRX2 receptor expressed on mast cells. EP262 is currently being evaluated in a mid-stage clinical trial for chronic spontaneous urticaria, a type of allergic skin condition. The acquisition is expected to strengthen Incyte’s position in the inflammatory disease space and provide potential launch opportunities starting in 2029.
