Patients who received a single gene-editing injection showed steady improvement in a genetic disorder that can lead to fatal swelling if left untreated. Wall Street, however, dismissed the good news.
Friday’s Phase 1 study results are encouraging news for genetic medicine and the treatment’s developer
(cue: NTLA)—but investors greeted it by plunging the company’s shares, along with those of others applying the Nobel-winning gene-editing technique known as Crispr-Cas9. Investors have clearly lost patience with the slow-moving clinical trials of gene therapies, and some are trying to take advantage of the upbeat news to exit their positions.
Intellia’s stock fell as much as 16% on Friday morning before settling down 7% by midday at $63.90, even though fans such as RBC Capital Markets analyst Luca Issi thought the early-stage study was good result.
Sales hit all Crispr medical developers.
(CRSP) saw its shares fall 7%.
The stock (EDIT) fell 11%. Caribou Biosciences (CRBU) fell 13%, while
fell 8%. Crispr gene editing treatments can make permanent changes to our DNA by targeting specific genes and then turning them off or rewriting harmful sections of their genetic instructions.
Asked about the Wall Street sell-off, an Intellia spokesman said the biotech can’t explain “every move in the share price.”
“But the news is clearly all good for Intellia and the patients Intellia hopes to serve.” the spokesman said.
Interim Phase 1 results announced by Intellia at a medical conference in Berlin on Friday morning provided the first data on a possible permanent solution for hereditary angioedema – a condition in which a miswritten gene in liver cells produces a protein that causes dangerous swelling all over the body.
In six patients, the one-time treatment reduced blood levels of the troublesome protein by more than 90%—a more profound effect than that achieved with chronic doses of the approved drug Takhzyro, developed by
(IONS) and is marketed by
(TAK). Edema attacks also decreased.
The angioedema results were on par with what RBC’s Issi said would be a “blue sky” result in an advance note on Thursday. Such a performance could lift Intellia shares above $85, he hoped. His 12-month price target is $150.
Talking to me Barron’s On Friday, after hearing from the company and investors, Issi said some of Friday’s drop in shares may reflect hedge funds “selling on the news,” after an earlier rally in Intellia shares on the expected good news. There was some relevant data – he notes – showing highly elevated liver enzymes in one patient. The effects have subsided, but investors are wary of new gene-editing treatments.
On Friday, Intellia also announced good long-term results in another Phase 1 study of a treatment it is developing in partnership with
). This Crispr injection neutralizes a rogue gene in liver cells, whose toxic output damages the heart or nerves to cause a disease known as transthyretin amyloidosis, or ATTR. In 12 patients, the treatment caused a more than 90% reduction in a poisonous protein produced by a rogue gene in their liver cells. Some patients were measured for six months, with results maintained.
Crispr’s unique treatment for ATTR appears on track to compete with chronic drug treatment Onpattro from
(ALNY), or the $2 billion-a-year Vyndaqel from
(PFE). Successful test results reported this year by Alnylam sent its stock soaring.
Regeneron stock was flat on Friday, despite a selloff in Intellia stock.
In a call Friday morning before the stock market opened, Intellia officials excitedly discussed their plans to move into controlled Phase 2 trials for the ATTR study. CEO John Leonard called the results a vindication of the company’s modular approach to Crispr therapies, whereby it can knock down different rogue genes by simply changing the guide-RNA piece in its products.
RBC’s Issi lamented in a note Friday that selling Intellia stock doesn’t make sense. “We are buyers because we believe [Intellia] it’s the best gene editing name in the space,” he said.
Write to Bill Alpert at firstname.lastname@example.org