Al­ny­lam shares dip on a de­ci­sion to scrap ac­cel­er­at­ed pitch on givosir­an; Loxo gets a 'break­through' at the FDA

Al­ny­lam $AL­NY has de­cid­ed to put a hold on its plans to shoot for an ac­cel­er­at­ed ap­proval of givosir­an and in­stead start a rolling sub­mis­sion in search of a full ap­proval. In­vestors didn’t care for what looked like a slow­down, dri­ving shares down by 1.8%. But at least one an­a­lyst was hold­ing fire this morn­ing. Stifel’s Paul Mat­teis not­ed: “This is not a sur­prise to us: this plan is, in our opin­ion, a byprod­uct of faster than ex­pect­ed en­roll­ment of the phase III EN­VI­SION tri­al, and we’d be buy­ing the stock on any weak­ness that en­sues.” This drug has been lined up as Al­ny­lam’s sec­ond RNAi drug in­tend­ed for the mar­ket. The drug is de­signed to treat acute he­pat­ic por­phyr­ia.

Sarep­ta Ther­a­peu­tics $SRPT has lined up an­oth­er gene ther­a­py for its pipeline. The Cam­bridge, MA-based biotech in-li­censed US rights to Lyso­gene’s pro­gram for San­fil­ip­po syn­drome type A, with the French com­pa­ny set to be­gin a piv­otal tri­al be­fore the end of this year. Sarep­ta is han­dling over $15 mil­lion for the re­gion­al rights to LYS-SAF302, with an­oth­er $2.5 mil­lion for an eq­ui­ty stake in the com­pa­ny. Next year they’ll fol­low up with an ad­di­tion­al $19 mil­lion, with mile­stones run­ning up to $125 mil­lion in the deal.

Loxo On­col­o­gy $LOXO is go­ing to get some help in its quest to hur­ry along the de­vel­op­ment of LOXO-292. The FDA has come through with a break­through drug des­ig­na­tion for its RET in­hibitor. Loxo’s drug is up against a ri­val from Blue­print Med­i­cines $BPMC. And now the pun­ters will give Loxo an edge in that show­down. The BTD pro­gram calls on reg­u­la­tors to keep an open-door pol­i­cy to a ther­a­py to help ac­cel­er­ate its pas­sage to the mar­ket. And it’s helped do that for a num­ber of can­cer drugs now be­ing sold.

Hi­FiBiO Ther­a­peu­tics has ac­quired H-Im­mune Ther­a­peu­tics, an ear­ly-stage biotech­nol­o­gy com­pa­ny and a spin-out of the French Atom­ic En­er­gy Com­mis­sion en­gaged in im­muno-on­col­o­gy R&D work.


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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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.

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.