Scott R. Grossman
Founder of Vindico Capital, Consumer-Sector Investor
uniQure's risk/reward is attractive because $10-12 of cash per share plus the upside implies the market assigns a low chance of approval. Management communication was the core issue rather than a change to the endgame; the unmet need is huge and the company is likely closer to the finish line than the start. The prespecified cUHDRS base and bull case was missed, possibly because of attrition, but being negative here is fundamentally betting the drug doesn't work. x.comÂ
uniQure is worth adding after a volatility-driven selloff that briefly valued the company near zero enterprise value. @A_May_MD notes the new four-year data is not part of the accelerated approval package, which rests on three-year cUHDRS data that improved; the four-year data missed significance on cUHDRS but stayed significant on TFC, the confirmatory endpoint, and the high dose still beats the low dose. The FDA's stance looks constructive, and an advisory committee will debate the data. x.com x.com x.comÂ
uniQure offers a much better risk-reward after today's news than before the November setback, as @LogicalThesis points out. As clarity on AMT-130's approval builds, it could return to or surpass its October level of about $70, though the market still has trust issues after what happened. If things turn negative again, the case is over. x.comÂ
uniQure's AMT-130 case looks stronger because, per Peter Mantas, Roche researchers reinforced the natural-history comparator argument. Mantas says objections to using Enroll-HD as a comparator are harder to defend ahead of a pivotal regulatory decision, and that the comparator is central to reimbursement. x.comÂ
uniQure's risk/reward is too high, as the stock has become a binary bet on regulatory and clinical outcomes. The earlier drop came because the FDA asked for a larger trial, a huge setback, yet the stock has since doubled as investors bet on success given very strong efficacy data. Future moves will hinge on regulatory and clinical catalysts, and the chart shows huge swings all the time. x.comÂ
uniQure's AMT-130 likely still gets approved and can be a reasonable commercial success. The open questions are insurance coverage and pricing, and whether Huntington's disease patients will pay given there is no other treatment. x.comÂ
ClearPoint Neuro has many irons in the fire beyond uniQure, one of its 60+ partners, which still has a decent probability of BLA acceptance in November. Neurona's MTLE drug and Parkinson's partners are enrolling, and gene and cell therapy delivered into the brain is not going to slow down. x.comÂ
uniQure and ClearPoint Neuro look very bullish on the Wall Street Journal report that President Trump is expected to remove FDA Commissioner Marty Makary. x.comÂ
ClearPoint Neuro would become a cash-flow machine if uniQure's AMT-130 reaches commercial launch, and after today's positive reversal it is a much better risk-reward than before the event, as @LogicalThesis points out. Commercial partners pay more for equipment and consumables than trial partners, and royalties flow straight to the bottom line. With more clarity on approval it could regain or pass its October 2025 peak near $30. Without an anchor commercial partner it is far less attractive. … Show more
Small- and mid-cap biotech looks like one of healthcare's more asymmetric setups after a four-year bear market, as AI speeds drug discovery and Big Pharma faces a patent cliff. Over $300B of pharma revenue goes off patent in the next few years, M&A is already ramping hard as pharma hunts for assets, and many small and mid-cap names still trade at reasonable valuations, making them potential takeover targets. x.comÂ
