FDA slaps a clin­i­cal hold on mes­sen­ger RNA pro­gram for rare ge­net­ic dis­ease

Trans­late Bio’s plans to put a sec­ond mes­sen­ger RNA drug in the clin­ic in the first half of 2019 has just ground to a halt in front of a sud­den reg­u­la­to­ry red light.

Ron Re­naud

The biotech $TBIO, which went pub­lic in the class of 2018, an­nounced this morn­ing that the FDA has slapped a clin­i­cal hold on its IND for MRT5201. The IV drug is de­signed to ad­dress rare cas­es of or­nithine tran­scar­bamy­lase (OTC) de­fi­cien­cy, an X-linked ge­net­ic dis­ease.

All we know right now is that the FDA has con­veyed ver­bal­ly that reg­u­la­tors have “ad­di­tion­al clin­i­cal and non­clin­i­cal ques­tions” for the biotech. The agency it­self nev­er dis­clos­es its po­si­tion on these cas­es pub­licly. 

Shares in the biotech was down 7.5% mid-day Tues­day — not a dread­ful out­come.

Leerink’s Ge­of­frey Porges talked with the man­age­ment crew, who gave the an­a­lyst the im­pres­sion that this hold was a lot like the tem­po­rary one they got for their lead ef­fort. But what­ev­er in­spired it, the FDA’s de­ci­sion has no im­pact on the top drug for cys­tic fi­bro­sis. He notes:

At this stage it is im­pos­si­ble to tell whether this clin­i­cal hold amounts to a short- or long-term de­vel­op­ment de­lay for MRT5201, but re­gard­less it does not seem to have any spillover im­pli­ca­tions for the com­pa­ny’s prin­ci­pal val­ue dri­ver, which is their lead de­vel­op­ment pro­gram MRT5005 in CF. The com­pa­ny as­sured us that en­roll­ment in the SAD phase of the MRT5005 pro­gram is con­tin­u­ing with­out change, and the com­pa­ny ex­pects to an­nounce the first CF pa­tient en­roll­ment in­to the MAD phase of this tri­al short­ly.

Mes­sen­ger RNA, a field led by a slate of biotechs in­clud­ing Mod­er­na and BioN­Tech, works by de­liv­er­ing code to a cell to in­struct it to make a drug. In this case the cod­ing di­rects liv­er cells to make nor­mal OTC en­zyme. That’s a con­cept that is easy to un­der­stand and dev­il­ish­ly dif­fi­cult to ac­com­plish. 

Trans­late, which is helmed by Ron Re­naud, says it is await­ing for­mal com­mu­ni­ca­tions from the FDA and then will tack­le what­ev­er is­sues are on the ta­ble. The com­pa­ny — which raised $122 mil­lion from its IPO and has a mar­ket cap of $250 mil­lion — has a Phase I/II tri­al un­der­way on cys­tic fi­bro­sis, ac­cord­ing to its web site.

Novartis CEO Vas Narasimhan [via Bloomberg/Getty]

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Vas Narasimhan has warily stepped up with what might pass as something close to a borderline apology for the latest scandal to engulf Novartis.

But he couldn’t quite get there.

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Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

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As Bloomberg notes, most of the Wall Street analysts that cover Sarepta $SRPT are an upbeat bunch, ready to cheer on the team when it comes to their Duchenne MD drugs, or offer explanations when an odd setback occurs — as happened recently with a safety signal that was ‘erroneously’ reported last week.

Ritu Baral Cowen
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Saqib Islam. CheckRare via YouTube

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SpringWorks and Satsuma — both biotech spinouts that have closed B rounds in April — are loading up with IPO cash to boost their respective late-stage plans.

Bain-backed SpringWorks is the better-known company of the two, and it’s gunning for a larger windfall of $115 million to add to $228 million from previous financings. In the process, the Stamford, CT-based team is also drawing the curtains on the partnerships it has in mind for the pair of assets it had initially licensed from Pfizer.

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Van­da shares slide af­ter FDA spurns their big end­point and re­jects a pitch on jet lag re­lief

Back in the spring of last year, Vanda Pharmaceuticals $VNDA served up a hot stew of mixed data for a slate of endpoints related to what they called clear evidence that their melatonin sleep drug Hetlioz (tasimelteon) could help millions of travelers suffering from jet lag.

Never mind that they couldn’t get a planned 90 people in the study, settling for 25 instead; Vanda CEO Mihael H. Polymeropoulos said they were building on a body of data to prove it would help jet-lagged patients looking for added sleep benefits. And that, they added, would be worth a major upgrade from the agency as they sought to tackle a big market.

Jim Mellon [via YouTube]

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On Monday, Juvenescence completed its $100 million Series B round of financing. The company is backed by British billionaire Jim Mellon — who wrote his 400-page guide to investing in the field of longevity shortly after launching the company in 2017. Bailey, who served as a board director for seven years at Medivation before Pfizer swallowed the biotech for $14 billion, is joined by Declan Doogan, an industry veteran with stints at Pfizer $PFE and Amarin $AMRN.

AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

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It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.