I spoke with Lauren Holder, a Huntington's patient and advocate, about this week's $QURE AMT-130 gene therapy clinical update. “I’m not surprised by the four-year results because this isn’t a cure,” Holder told me. “This is a slowing-of-disease-progression treatment, and so I would expect there’s still decline going on, and we’re going to see that on testing, and that doesn’t mean it’s not working.” Holder is a lot less pleased with the proposed design of UniQure's confirmatory study, which will require some patients to be randomized to a standard treatment arm. "It’s not ethical,” she said. “You’re requiring patients to not receive any treatments for three years or longer, and to not be able to participate in other research. To me, it’s not ethical. To the community, it’s not ethical, and I really feel we need to have a better option.” Read more:

STAT sent @Jasonmmast to Shanghai to report first hand on China's booming biotech scene. In Shanghai’s ‘Science City,’ China’s ascendant biotechs make their pitch to the world

Last March, $IMMX 's management team and board hired a new chief medical officer who was living under a fake name after spending 20 years as a fugitive following a conviction for sexual assault. Yesterday, the same Immix executives turned a positive study readout into an 8% drop in the stock price because of a clumsy financing and an amateur-hour conference call. See a pattern here?

So. Announcements about #STATSummit#, which is happening in Boston on less than a month. Stephen Hoge from $MRNA and Dean Li from $MRK will be joining us to talk about the companies cancer vaccine, which they have said dramatically slowed the return of cancer in patients with metastatic melanoma. That will be one of many sessions on cancer. We'll have patient stories on the Merck-Moderna vaccine, on Rasonque, and other targeted drugs. And we'll feature a discussion of top oncologists including Robert Winn and Brian Wolpin, who presented the Rasonque data at ASCO. I also want to note here that Tony Fauci has canceled his appearance at the summit.

Obviously, the news that $BNTX and Roche are stopping their cancer "vaccine" study in adjuvant colorectal cancer is not good. It's also not that big a surprise. This is a reminder that this technology won't work in every cancer and may work best in the areas where medicines like Keytruda are already effective. Keytruda is only used in colorectal cancer when it is driven by defects in DNA repair (MSI-High or mismatch repair deficient cancer). So this was a longer-shot trial. The one to watch for the $BNTX is pancreatic cancer. The biggest market for such treatments -- and the one valuations will swing on -- is non-small cell lung cancer, which is also the big market for Keytruda. This is probably less of a big deal than it seems at first glance. $BNTX $ARCT $MRNA $MRK are all down.

$ALNY -5% this morning Sellside comments re: the 1.14 event rate ratio from the stabilizer (combo) arm of the CARDIO-TTRansform study: Paul Matteis, Stifel "While minimal benefit was largely expected for the eplontersen/tafamidis combo arm in CARDIO-TTRansform, it's an incremental surprise to see that the combination had a hazard ratio of 1.14--clearly worse than expected." Michael Leuchten, Jefferies: "The presentation showed a treatment effect of rate ratio (RR) 1.14 for the baseline stabiliser group vs overall RR of 0.89, with a strong enough interaction p-value (0.017) suggesting a potential differential treatment effect between patients without stabiliser at baseline. The primary endpoint curves in patients that were on stabilisers at baseline do not show an improvement over time, if anything there is a worsening rate in the combination group." Cory Kasimov, Evercore: "...the 1.14 RR in pts on baseline stabilizers was the key metric investors cared about most coming in and will likely not be viewed favorably as it relates to implications for ALNY. At first glance, the results appear less suggestive of a trial-design issue and more consistent with a potential limitation to the incremental benefit of dual therapy with silencers and stabilizers, further entrenching the competitive positioning of the stabilizer class."