The field of multiple myeloma has transformed from having few treatments to an abundance of effective drugs. The primary clinical challenge is no longer finding a therapy that works, but rather determining the optimal sequence and combination of available options, highlighting a unique form of market maturity.
The disruption to the U.S. biomedical research ecosystem is not necessarily a targeted reform of science itself. Instead, it's viewed by many as 'collateral damage' in a larger political culture war against universities and perceived 'woke leftist ideologies,' with NIH funding being used as leverage.
Terns Pharmaceuticals, previously known for its obesity and MASH programs, scored a major win with its chronic myeloid leukemia drug. This success demonstrates that a diversified, seemingly unfocused early-stage pipeline can be a strategic asset, allowing a company to pivot to a surprise blockbuster when other programs fail.
The Trump administration's actions have eroded the long-standing trust that the federal government will provide stable, long-term research funding. This breakdown of the 'social contract' discourages scientists from pursuing ambitious, multi-decade longitudinal studies, which are crucial for major breakthroughs but are now perceived as too risky.
Despite showing massive weight loss, new obesity drugs from Eli Lilly and others have high discontinuation rates due to side effects. This suggests the industry's singular focus on efficacy may be hitting diminishing returns, opening a new competitive front based on better patient tolerance and adherence.
Despite the landmark approvals of two complex gene therapies for sickle cell disease, their commercial rollout has been slow. An effective, easy-to-administer pill from Fulcrum Therapeutics could completely disrupt the market by offering a simpler, more accessible alternative, demonstrating how 'good enough' technology can beat a more complex breakthrough.