Gene-editing startups have captured the attention—and wallets—of Wall Street.
They’re powered by the Nobel Prize-winning technique known as Crispr-Cas9, and an important test of the technology’s value is coming Saturday.
The treatment, developed by Intellia with Regeneron’s backing, turns off a mutant gene that drives liver cells to make a harmful protein that damages the heart and nerves. The disease, known as transthyretin amyloidosis, or ATTR, is treatable now only with drugs.
Encouraging numbers from the Phase 1 trial would vindicate the investors who bid up Intellia stock (ticker: NTLA) some 275% in the past year. By comparison, the Nasdaq Composite rose about 40% and Regeneron (REGN) lost 17% as excitement about its Covid-19 antibody products waned.
is expected to surpass $2 billion this year with sales of its Vyndaqel and Vyndamax, which alleviate some damage. Alnylam’s $173 stock price and $20 billion market capitalization are largely underpinned by its fast-growing Onpattro and Vutrisiran, which can greatly slow and—in some cases—even halt progression of the disease.
“This will be a big catalyst for the whole field of gene editing,” said Luca Issi, an analyst at RBC Capital Markets who is betting the data will be positive. He launched coverage of Intellia in May with an Outperform rating.
Issi prefers Intellia over peers
(EDIT) because of its lead in editing genes inside the body. Editas actually launched the first clinical trial of an in-body technique, but is targeting a rare eye disorder in its first clinical trial and won’t report its data until later this year. Crispr Therapeutics has treated more patients in its clinical trials than both Intellia and Editas, but those therapies— for cancer and sickle-cell disease—are performed outside the body: A patient’s cells are harvested, edited in a lab, then reinfused.
For Issi, Intellia needs to deliver results that show its therapy is at least 75% effective in eliminating ATTR’s harmful protein. If the rate is that high, he thinks the treatment will be judged a worthy alternative to current drugs. Infusions of Alnylam products can reduce blood levels of the harmful protein by 80% to 90%. Pfizer’s pills render some of the ATTR proteins harmless, thereby slowing the damage done by the disease.
If Intellia delivers an impressive number, Issa thinks the stock could jump from recent levels below $80 to above $95. A successful one-time treatment could be priced above $1 million, Issa believes, with annual sales surpassing $2 billion a decade from now. If investors come around to his way of thinking, the analyst sees Intellia stock hitting $110 within a year.
Guggenheim’s Debjit Chattopadhyay thinks Intellia has to match or top Alnylam’s 80% to pose a real challenge.
Alnylam’s drugs have become the standard of care, writes Chattopadhyay, who bought Alnylam stock from 2018 until February 2021, when the stock’s $162 price shifted his rating to Neutral. Alnylam has said it is developing formulations that would have to be infused only twice, or even once a year.
And, of course, Alnylam’s drug treatments are reversible. If safety problems emerge with gene-editing, their effects may be hard to reverse.
Write to Bill Alpert at [email protected]