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Msg  42678 of 43135  at  9/22/2022 12:56:02 PM  by

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Intellia Investors Are Dumping Stock on News of Crispr Gene-Editing Success

Intellia Investors Are Dumping Stock on News of Crispr Gene-Editing Success
Bill Alpert / Barrons Sept 16, 2022

Patients who got a one-time gene-editing infusion showed sustained improvement in a genetic disorder that can leads to fatal swelling, when untreated. Wall Street, however, spurned the good news.

Friday's Phase 1 study results are great news for genetic medicine and for the treatment developer Intellia Therapeutics (NTLA) -- but investors greeted it by dumping the company's stock, along with those of others applying the Nobel-prize winning technique for altering genes that's known as Crispr-Cas9. Investors have clearly lost patience with the slow-paced clinical trials of genetic therapies, and some are trying to take advantage of the upbeat news to get out of their positions.

Intellia stock is down 9% Friday morning, to $62.64, even though admirers such as RBC Capital Markets analyst Luca Issi considered the early-phase study a great outcome.

Selling hit all the Crispr medicine developers. CRISPR Therapeutics (CRSP) saw a 7% drop in its shares. Editas Medicine (EDIT) stock fell 11%. Caribou Biosciences (CRBU) dropped 12%, while Beam Therapeutics fell 10%. Crispr gene- editing treatments can make permanent changes in our DNA by homing in on specific genes and then disabling them or rewriting harmful sections of their genetic instructions.

Intellia didn't immediately respond to Barron's request for comment on Wall Street's selling.

The study results announced by Intellia at a Berlin medical conference Friday morning could hardly have been better. The Phase 1 trial gave a single infusion to each of six patients who suffer from hereditary angioedema, a condition in which a miswritten gene in their liver cells produces a protein that causes dangerous swelling throughout the body. The one-time treatment reduced blood levels of the troublesome protein by over 90% -- a more-profound effect than achieved by chronic doses of the approved drug Takhzyro, developed by Ionis Pharmaceuticals (IONS) and marketed by Takeda Pharmaceutical (TAK).

The angioedema results were as good as what RBC's Issi said would be a "blue sky" outcome in an advance note on Thursday. Such a showing could lift Intellia shares above $85, he had hoped. His 12-month target price is $150.

In a Friday note, Issi surmised that investors might have been spooked by elevated liver enzymes in one patient, but no symptoms resulted and the affects subsided.

On Friday, Intellia also announced good longer-term results in another Phase 1 study of a treatment it's developing in partnership with Regeneron Pharmaceuticals ( REGN). This Crispr infusion knocks out a rogue gene in liver cells, whose poisonous output damages the heart or nerves to cause a disease known as transthyretin amyloidosis, or ATTR. In 12 patients, it caused a more than 90% drop in a poisonous protein made by a rogue gene in their liver cells. Some patients have been measured for six months, with the results sustained.

The one-and-done Crispr treatment for ATTR looks on track to rival the chronic drug treatment Onpattro from Alnylam Pharmaceuticals (ALNY), or the $2 billion-a-year Vyndaqel from Pfizer (PFE). Successful trial results reported this year by Alnylam had led to a pop in its stock.

Regeneron stock is flat Friday, despite the selling in Intellia's stock.

On a Friday morning call before the stock market opened, Intellia officials happily discussed their plans to advance to Phase 2 controlled trials. 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 bit of guide-RNA on its products.

RBC's Issi lamented in his Friday note that the Intellia stock selloff makes little sense. "We are buyers given we believe that [Intellia] is the best gene-editing name in the space," he declared.


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