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]

I’m not per­fect: No­var­tis chief Vas Narasimhan al­most apol­o­gizes in the wake of a new cri­sis

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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Af­ter a posse of Wall Street an­a­lysts pre­dict a like­ly new win for Sarep­ta, we're down to the wire on a crit­i­cal FDA de­ci­sion

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

Spring­Works seeks $115M to push Pfiz­er drugs across fin­ish line while Sat­suma sells mi­graine play in $86M IPO

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.
SpringWorks

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.

Mi­nor­i­ty racial groups con­tin­ue to be dis­mal­ly rep­re­sent­ed in can­cer tri­als — study

Data reveal that different racial and ethnic groups — by nature and/or nurture — can respond differently in terms of pharmacokinetics, efficacy, or safety to therapeutics, but this disparity is not necessarily accounted for in clinical trials. A fresh analysis of the last decade of US cancer drug approvals suggests the trend continues, cementing previous research that suggests oncology trials are woefully under-representative of the racial makeup of the real world.

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]

Health­i­er, longer lifes­pans will be a re­al­i­ty soon­er than you think, Ju­ve­nes­cence promis­es as it clos­es $100M round

Earlier this year, an executive from Juvenescence-backed AgeX predicted the field of longevity will eventually “dwarf the dotcom boom.” Greg Bailey, the UK-based anti-aging biotech’s CEO, certainly hopes so.

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

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

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.