Translate Bio Drops Liver- Disease Program. The Stock Is Falling.

Josh Nathan-Kazis Health MRNA TBIO Moderna Translate Bio Pharmaceuticals Health Care/Life Sciences Ronald Renaud Geoffrey Porges Josh Schimmer Renaud Political/General News Health Genetic Disorders Medical Conditions I/DRG M/HCR N/DJN N/GEN N/HLT N/WER Barrons.com Barrons Blogs Wires CODES_REVIEWED Health author Josh Nathan-Kazis author|Josh Nathan-Kazis topicid 8583 name Josh Nathan-Kazis extractedtext Josh Nathan-Kazis rank 1 codetype author code josh_nathan_kazis nameformat surname_first author Josh Nathan-Kazis id Josh Nathan-Kazis barrons_display_subject BARHEALTH barrons_display_subject|BARHEALTH codetype BARRONS_DISPLAY_SUBJECT canbedisplaysubject true value BARHEALTH source MANUAL status modified name Health code BARHEALTH co WHXDNB co|WHXDNB symbol TBIO country US codetype co displayname Translate Bio extractedtext Translate Bio source MANUAL code tbio name Translate Bio significance prominent onlinesignificance prominent exchange U.S.: Nasdaq exchangeisocode XNAS chartingsymbol STOCK/US/XNAS/TBIO fcode WHXDNB status modified co MDRTHU co|MDRTHU symbol MRNA country US codetype co displayname Moderna extractedtext Moderna source MANUAL code mrna name Moderna significance prominent onlinesignificance passing-mention exchange U.S.: Nasdaq exchangeisocode XNAS chartingsymbol STOCK/US/XNAS/MRNA fcode MDRTHU status modified company TBIO company|TBIO name Translate Bio significance PROMINENT company MRNA company|MRNA name Moderna significance PASSING-MENTION djn I/DRG djn|I/DRG significance prominent onlinesignificance prominent name I/DRG why about source FACTIVA fcode I/DRG codetype djn code i_drg djn M/HCR djn|M/HCR name M/HCR why lineage source FACTIVA fcode M/HCR codetype djn code m_hcr djn N/GEN djn|N/GEN name N/GEN why lineage source FACTIVA fcode N/GEN codetype djn code n_gen djn N/HLT djn|N/HLT name N/HLT why lineage source FACTIVA fcode N/HLT codetype djn code n_hlt djn N/DJN djn|N/DJN codetype djn value N/DJN source DJN-CONTROL status modified inactivebydefault true name N/DJN code n_djn djn N/WER djn|N/WER codetype djn value N/WER source DJN-CONTROL status modified inactivebydefault true name N/WER code n_wer first_publish_headline Translate Bio Drops Liver- Disease Program. The Stock Is Falling. first_publish_headline|Translate Bio Drops Liver- Disease Program. The Stock Is Falling. flow NWREGULAR flow|NWREGULAR codetype FLOW value NWREGULAR source MANUAL status modified name Wires code NWREGULAR flow Barrons.com flow|Barrons.com codetype FLOW value Barrons.com source MANUAL status modified name Barrons.com code online name Online title Product code Barrons.com flow Barrons Blogs flow|Barrons Blogs codetype FLOW value Barrons Blogs title Product source MANUAL status modified name Barrons Blogs code Barrons_Blogs headline Translate Bio Drops Liver- Disease Program. The Stock Is Falling. headline|Translate Bio Drops Liver- Disease Program. The Stock Is Falling. in i257 in|i257 significance prominent onlinesignificance prominent name Pharmaceuticals why about source FACTIVA fcode i257 codetype in code i257 in i951 in|i951 name Health Care/Life Sciences why lineage source FACTIVA fcode i951 codetype in code i951 media media-2 media|media-2 responsive layout wrap softcrop Horizontal imphotoid im-102092 location https://images.barrons.com/im-102092/?size=1.5 media media-1 media|media-1 softcrop Horizontal imphotoid im-102092 location https://images.barrons.com/im-102092/?size=1.5 ns ghemo ns|ghemo significance prominent onlinesignificance prominent name Genetic Disorders why about source FACTIVA fcode ghemo codetype ns code ghemo ns gcat ns|gcat name Political/General News why lineage source FACTIVA fcode gcat codetype ns code gcat ns ghea ns|ghea name Health why lineage source FACTIVA fcode ghea codetype ns code ghea ns gmed ns|gmed name Medical Conditions why lineage source FACTIVA fcode gmed codetype ns code gmed nwchain SB527738632899569448639045855420725950754641 nwchain|SB527738632899569448639045855420725950754641 pe 132541291 pe|132541291 lastname Renaud displayname Renaud, Ronald confidence 93 why occur extractedtext Ronald Renaud source FACTIVA relevance 50 significance passing firstname Ronald onlinesignificance passing-mention name Ronald Renaud confidencerange high relevancerange low fcode 132541291 codetype pe code 132541291 nameformat surname_first pe Geoffrey Porges pe|Geoffrey Porges firstname Geoffrey lastname Porges displayname Porges, Geoffrey name Geoffrey Porges extractedtext Geoffrey Porges source FACTIVA codetype pe code geoffrey_porges nameformat surname_first pe Josh Schimmer pe|Josh Schimmer firstname Josh lastname Schimmer displayname Schimmer, Josh name Josh Schimmer extractedtext Josh Schimmer source FACTIVA codetype pe code josh_schimmer nameformat surname_first pe Renaud pe|Renaud firstname Renaud displayname Renaud name Renaud extractedtext Renaud source FACTIVA codetype pe code renaud nameformat surname_first relay SYND relay|SYND name Syndication source EXPANDER value SYND codetype RELAY code synd status modified statistic CODES_REVIEWED statistic|CODES_REVIEWED name CODES_REVIEWED value CODES_REVIEWED codetype STATISTIC code CODES_REVIEWED subject BARHEALTH subject|BARHEALTH ruleid BARHEALTH codetype SUBJECT value BARHEALTH canbedisplaysubject true name Health title Health status modified code BARHEALTH wordcount 674 wordcount|674 Courtesy of Translate Translate Bio Drops Liver- Disease Program. The Stock Is Falling. Translate Bio Drops Liver- Disease Program. The Stock Is Falling. Translate Bio Drops Liver-Disease Program, and Stock Falls

Shares fell more than 8% in premarket trading. The company said it was halting the development program in light of disappointing preclinical studies.

Translate Bio Drops Liver- Disease Program. The Stock Is Falling.

Shares fell more than 8% in premarket trading. The company said it was halting the development program in light of disappointing preclinical studies.

https://investors.translate.bio/news-releases/news-release-details/translate-bio-announces-pipeline-program-update https://www.barrons.com/articles/mrna-is-the-next-frontier-in-treating-disease-but-will-it-work-51567205845 mailto:josh.nathan-kazis@barrons.com Translate Bio Drops Liver- Disease Program. The Stock Is Falling. By Josh Nathan-Kazis Courtesy of Translate Translate develops therapies based on synthetic mRNA.

Translate Bio, a clinical-stage biotech company developing therapies based on synthetic mRNA, said Monday night that it was dropping one of its main drug-development programs, an effort to treat a liver disease called ornithine transcarbamylase deficiency.

The stock fell 8.3% in premarket trading on Tuesday. Translate (ticker: TBIO) said it was dropping the program in light of disappointing preclinical studies of the OTC deficiency drug, and in order to prioritize the company’s cystic fibrosis program, which has produced promising clinical results.

“We believe that the success to date in our cystic fibrosis program positions us well to build on our lung delivery platform and maximize the potential of our mRNA technology in additional pulmonary diseases with unmet medical need,” the company’s CEO, Ronald Renaud, said in a statement.

Evercore ISI analyst Josh Schimmer, who rates the company Outperform, dropped his price target to $14 from $20 on the news after removing the OTC program from his model. “It’s all a little unsettling in a way we are still trying to articulate,” Schimmer wrote.

The back story. Translate Bio is one of a handful of companies seeking to use messenger RNA as a drug, and was one of the subjects of a Barron’s cover story on the technology in August. It is the smaller of the two public companies specializing in the technology, after Moderna (MRNA). As of the end of trading on Monday, Translate’s stock was up 33.1% since the start of the year.

What’s new. In its statement Monday night, Translate said it was terminating its preclinical OTC deficiency program “based on data from recently completed preclinical studies which do not support the desired pharmacokinetic and safety profile for advancement of the program.” The drug has not yet been tested in people.

The OTC deficiency treatment that Translate was testing used a different delivery vehicle than the company’s cystic fibrosis treatment. Both are so-called lipid nanoparticles, bundles of fats that protect the synthetic mRNA as it travels into the cell, but each is constituted differently. Translate said that the disappointing data on the OTC deficiency drug was related to that drug’s lipid nanoparticle, which was designed to target liver diseases.

The company said that it would now prioritize its pulmonary-disease work, noting the promising preliminary data produced by a continuing ongoing Phase 1/2 study of its cystic fibrosis treatment.

In a note out Tuesday, SVB Leerink analyst Geoffrey Porges maintained his Outperform rating and his $20 price target on the stock, saying he had already removed the OTC program from his model.

“It is encouraging to see TBIO commit to this platform and the CF program and is consistent with the groundbreaking potential of its lung LNP delivery system,” Porges wrote. “The decision to abandon the OTCD program was probably relatively easy given the challenges associated with that indication for TBIO and other developers.”

Looking forward. The company is currently testing its cystic fibrosis drug in a human trial; additional data is expected next year. Meanwhile, the company said it continues to work to develop new lipid nanoparticles.

“We design each LNP with distinct chemical structures and formulations specific to the target organ and route of administration in order to optimize safety and potency,” Renaud said in a statement. “We are excited about the robust mRNA delivery effort underway at Translate Bio and we’ll continue to apply our expertise in the development of novel lipids.”

Write to Josh Nathan-Kazis at josh.nathan-kazis@barrons.com

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.