Dr J Rould
Full-Time Tech Investor, Quantum/Cybersecurity, Philosophy Background
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
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
Mirum's small indications cap its upside, while Q4 Hep D phase 3 data must be good and its PSC filing on phase 2 data risks rejection after the FDA asked for phase 3. Those risks are priced in only below $80, where Mirum is worth holding and building. Above $95 it is an easy sell until the risks play out; $99 is a stated sale level if the stock gets too expensive. x.com x.comÂ
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
Ionis is best avoided because major Phase 3 trials still hang over it, making it hard to size a meaningful position while controlling risk. Biotechs can drop 10-30% overnight on a negative read-through from another company's similar drug, with no chance to react, and a 1-2% position is mostly a waste of time. Biotech names are worth owning only once profitable, generating real cash and free of major Phase 3 risk. x.comÂ
Taysha Gene Therapies is a high-conviction binary bet: cash cushions the downside, and FDA approval of TSHA-102 for Rett syndrome could be a 5-10x event. No disease-modifying Rett therapy exists today, and TSHA-102 has a clean safety record in 12 patients, while rival Neurogene's NGN-401 lost its high-dose arm after a patient death. Comparables suggest $2-4 million a dose against about $10,000 in cost. Cash of $319.8 million lasts into 2028, but another raise is likely; failure means near zero. … Show more
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
Intellia Therapeutics has bad management and challenged science, and it would not be surprising to see it go bankrupt after its commercial sales fail to live up to the hype, perhaps even before then. Avoiding big disasters like this matters more than finding a million great ideas, so losers should be cut quickly. 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 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
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
