After a decade of development and multiple clinical setbacks, Sanofi's venglustat has met its primary goal in a Phase 3 trial for Type 3 Gaucher disease, offering new hope for neurological symptoms. The drug, however, failed a separate late-stage study for Fabry disease.
Sanofi's experimental oral drug venglustat demonstrated significant neurological benefits for patients with Type 3 Gaucher disease in a late-stage trial, but failed to meet its primary endpoint in a separate study for Fabry disease, casting uncertainty on its regulatory path.
Bank of America has upgraded Apellis Pharmaceuticals (APLS) to 'Buy,' citing robust early adoption of its drug Empaveli and significant undervaluation of its potential in treating rare kidney diseases, despite a more cautious outlook from Wells Fargo.
Idorsia's exclusive licensing agreement with EMS to commercialize insomnia drug QUVIVIQ across Latin America marks a key step in its global expansion. The deal, involving $20 million in milestones and royalties, arrives as long-term data for its Fabry disease candidate lucerastat puts the spotlight on the company's broader late-stage pipeline and persistent financial pressures.
Sanofi reports a pivotal Phase 3 win for its investigational therapy venglustat in a rare neurological disorder, alongside positive EU regulatory news for Rezurock and progress for amlitelimab, signaling a strategic shift toward high-value specialty medicines.
Barclays' upgrade of PTC Therapeutics to Overweight has spotlighted the biotech firm's valuation, with shares showing strong momentum but questions lingering over its path to profitability and reliance on a narrow drug portfolio.
Vera Therapeutics (VERA) is drawing bullish attention from Wall Street, with multiple analysts significantly raising price targets following a key competitor's FDA approval and revised revenue forecasts for its lead drug candidate.
Jefferies has downgraded Amicus Therapeutics to 'Buy' from 'Hold' and trimmed its price target to $14.50, aligning it with BioMarin's pending $4.8 billion acquisition offer. The move reflects analyst skepticism about competing bids emerging for the rare disease biotech firm.
Shares of clinical-stage biotech Scholar Rock (NASDAQ: SRRK) have surged nearly 60% in three months, closing at $44.34. This rally, backed by promising clinical progress, has investors weighing its premium valuation against its long-term potential in treating serious diseases.
Barclays initiates coverage with an Overweight rating and $20 price target on Altimmune, citing strong fundamentals and pipeline potential. The biotech firm prepares to channel recent offering proceeds toward its pivotal Phase 3 development for metabolic liver disease treatment pemvidutide.
Pharmaceutical giant Eli Lilly enters a strategic collaboration potentially worth up to $1.93 billion with biotech firm Repertoire Immune Medicines, aiming to develop precision treatments for autoimmune diseases that move beyond broad immunosuppression.
Cogent Biosciences secures a key FDA Breakthrough Therapy Designation for its bezuclastinib-sunitinib combination in advanced GIST, following promising Phase 3 data that showed a halving of disease progression risk.
New long-term results from the pivotal EMBARK trial indicate ELEVIDYS, Sarepta's gene therapy, continues to alter the disease trajectory for boys with Duchenne muscular dystrophy, with no new safety concerns emerging over three years.
Reed Jobs, son of the late Apple co-founder Steve Jobs, has secured over $200 million for Yosemite, a venture fund dedicated to pioneering cancer treatments. Backed by major institutions, the fund seeks to transform cancer from a fatal diagnosis to a chronic, manageable disease within a generation.
Novo Nordisk charts a bold new course, launching a pivotal oral obesity drug while restructuring its workforce and deepening its investment in next-generation cell therapies for metabolic diseases.
Novo Nordisk launches its first oral GLP-1 weight-loss drug with strong early demand, reports positive Phase 3 results for CagriSema, and expands a cell therapy partnership for diabetes. These moves come as investors reevaluate the company's long-term growth prospects amid competitive and pricing pressures.
A coalition of environmental groups is sounding the alarm over two massive Royal Caribbean developments in Cozumel and Mahahual, warning of severe ecological strain and social disruption, as legal challenges begin to mount.
The White House announces a major public-private initiative to stockpile rare earths and critical minerals, aiming to insulate U.S. defense and tech sectors from foreign supply shocks and geopolitical leverage.
French pharmaceutical giant Sanofi has discontinued development of its experimental mRNA flu vaccine, citing a strategic reprioritization. The decision comes as influenza activity remains high across much of the United States, with significant hospitalizations and deaths reported this season.
Spanish healthtech startup Biorce has closed a substantial Series A round, backed by prominent venture capital and angel investors, to scale its AI-driven clinical trial solution, Aika, internationally amid a tightening regulatory landscape.
