IONS, AZN Stocks Fall On Full Data From Failed Cardiomyopathy Trial — Jefferies Sees Neutral To Negative Readthrough To ALNY

Ionis and AstraZeneca already disclosed on July 9 that the late-stage CARDIO-TTRansform missed its primary goal. Full data from the trial was unveiled on Friday.

• The failed trial studied Wainua in transthyretin amyloid cardiomyopathy or ATTR-CM. 
• Jefferies said in a note that the data presents a neutral to negative read-through to Alnylam, which is developing drugs for ATTR amyloidosis.
• Jefferies said the results were mostly as expected and put more pressure on Alnylam’s TTR business.

Shares of Alnylam Pharmaceuticals (ALNY) closed flat on Friday after full results from a failed cardiomyopathy trial conducted by Ionis Pharmaceuticals and AstraZeneca were presented in Munich.

Jefferies said in a note that the data presents a neutral to negative readthrough to Alnylam, which is developing drugs for ATTR amyloidosis, targeting the same protein as Ionis and AstraZeneca’s eplontersen.

Alnylam shares fell as much as 5% but closed Friday marginally higher. Ionis closed down 2%, and AstraZeneca’s U.S. shares slipped about 1%.

What Happened?

Ionis and AstraZeneca already disclosed on July 9 that the late-stage CARDIO-TTRansform missed its primary goal. Friday’s ESC Congress Hot Line session and a simultaneous New England Journal of Medicine paper supplied the first complete data.

The 1,432-patient study is the largest trial to date in transthyretin amyloid cardiomyopathy or ATTR-CM, a progressive and often fatal disease in which a misfolded protein builds up in the heart. Patients received a monthly injection of eplontersen (Wainua) or placebo for 140 weeks, on top of usual care. Wainua is co-developed and commercialized by Ionis and AstraZeneca.

The main measure in the trial combined cardiovascular deaths and repeat cardiovascular events. There were 381 events in 210 patients on eplontersen versus 392 events in 231 patients on placebo. The difference was not statistically significant.

The drug lowered circulating transthyretin as intended. Patients already on a stabilizer received no additional benefit. Those not on a stabilizer at the start had fewer events with eplontersen alone, a result that was only nominally significant. Safety was in line with earlier studies.

Wainua remains approved for nerve damage caused by a rare, inherited disease where abnormal amyloid proteins build up in the body, called hereditary transthyretin-mediated amyloidosis.

Wall Street Weighs In On Impact For ALNY

Jefferies said the results were mostly as expected and put more pressure on Alnylam’s TTR business — its drugs for ATTR amyloidosis, a disease in which a misfolded liver protein builds up in the heart or nerves. Alnylam’s main drug, Amvuttra, is a silencer that tells the liver to make less of that protein. The data raise doubts that silencers should be the usual next step after stabilizer pills already used as standard care, and they add risk to TRITON-CM, Alnylam’s late-stage trial of its next silencer in heart patients.

The firm kept a Hold rating and a $230 price target on ALNY.

How Did Stocktwits Users React?

On Stocktwits, retail sentiment around ALNY remained in ‘bearish’ territory over the past 24 hours, while message volume rose from ‘low’ to ‘normal’ levels.

Sentiment around IONS and AZN remained ‘neutral’ at the time of writing.

While ALNY stock has fallen 41% year-to-date, AZN has fallen 7% and IONS 23%. 

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