FDA hits a green light on 2 more rare dis­ease drugs, giv­ing Alex­ion an ear­ly shot at pro­tect­ing PNH fran­chise

Faced with the prospect of a par­tial gov­ern­ment shut­down lat­er to­day, the FDA has pulled the trig­ger on a pair of new drug ap­provals.

Alex­ion got its nod 2 months ear­ly for ALXN1210 for parox­ys­mal noc­tur­nal he­mo­glo­bin­uria, or PNH, which will now be sold as Ul­tomiris. And Stem­line Ther­a­peu­tics won an FDA ap­proval to mar­ket El­zon­ris (tagrax­o­fusp-erzs) for rare cas­es of blas­tic plas­ma­cy­toid den­drit­ic cell neo­plasm, or BPD­CN.

The FDA has now ap­proved 59 new drugs through CDER in 2018, part of its record-set­ting year as the in­dus­try en­joys a surge of new mar­ket­ing OKs.


Ge­of­frey Porges

Alex­ion $ALXN is plan­ning to price Ul­tomiris at a dis­count to Soliris, long one of the most ex­pen­sive ther­a­pies in the world. ALXN1210 proved just as ef­fec­tive as their big drug Soliris in treat­ing PNH, with less fre­quent dos­ing that will now pave the way to a dis­count price. Leerink’s Ge­of­frey Porges counts him­self as a sup­port­er of this strat­e­gy ahead of prospec­tive com­peti­tors for their PNH fran­chise.

While in­vestors may re­act neg­a­tive­ly to the ini­tial im­pact of this pric­ing head­wind (rev­enue for the PNH in­di­ca­tion for es­tab­lished pa­tients down 10%), we be­lieve the stock al­ready dis­counts sub­stan­tial price ero­sion and mar­ket share loss to com­peti­tors. In­stead, Alex­ion’s de­ci­sion to move to­ward more sus­tain­able pric­ing with a su­pe­ri­or prod­uct should front-run and counter these po­ten­tial risks, and build to­ward a more sta­ble rev­enue tra­jec­to­ry and longevi­ty than cur­rent­ly ex­pect­ed. 

Stem­line $STML, mean­while, had to en­dure a ker­fuf­fle ear­li­er on re­gard­ing its tardy ac­knowl­edge­ment of a pa­tient death dur­ing a small study of their drug, but the agency nonethe­less pro­vid­ing a thumbs-up on a small set of da­ta.

The drug was waved through af­ter post­ing wide­ly vary­ing re­sults from two pa­tient co­horts which re­ceived the drug in a tiny sin­gle-arm study.

The ef­fi­ca­cy of El­zon­ris was stud­ied in two co­horts of pa­tients in a sin­gle-arm clin­i­cal tri­al. The first tri­al co­hort en­rolled 13 pa­tients with un­treat­ed BPD­CN, and sev­en pa­tients (54%) achieved com­plete re­mis­sion (CR) or CR with a skin ab­nor­mal­i­ty not in­dica­tive of ac­tive dis­ease (CRc). The sec­ond co­hort in­clud­ed 15 pa­tients with re­lapsed or re­frac­to­ry BPD­CN. One pa­tient achieved CR and one pa­tient achieved CRc.

The OK comes with a black box warn­ing alert­ing physi­cians to be on alert for a host of po­ten­tial side ef­fects, but this is one con­di­tion where there are no ap­proved drugs, putting it on a pri­or­i­ty list for the FDA, which of­fered its break­through drug des­ig­na­tion to help speed it along.

Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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San Diego cou­ple charged with steal­ing trade se­crets, open­ing Chi­nese biotech as DOJ crack­down con­tin­ues

A San Diego couple has been charged with stealing trade secrets from a US hospital and opening a business based off those secrets in China as the controversial industry-wide crackdown on alleged corporate espionage continues. On the same day, the Department of Justice announced they had arrested Beijing representative Zhongsan Liu for allegedly trying to obtain research visas for government recruiters.

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Deborah Dunsire. Lundbeck

UP­DAT­ED: Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time Monday morning the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation of a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month. In a followup call, Dunsire also noted that the company will likely need 200 to 250 reps for this marketing task on both sides of the Atlantic. And analysts were quick to note that the dealmaking at Lundbeck isn’t done, with another $2 billion to $3 billion available for more deals to beef up the pipeline.

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Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.

UP­DAT­ED: Bio­gen pulls the plug on prized IPF drug from $562M+ Stromedix buy­out

One of Biogen’s attempts to branch out has flopped as the biotech scraps a mid-stage program for idiopathic pulmonary fibrosis.

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Warts for the win: Aclar­is' lead drug clears piv­otal study

Aclaris Therapeutics has found a way to get rid of the warts and all.

The company — which earlier this month decided to focus on its arsenal of kinase inhibitors — on Monday unveiled positive data from a pivotal study testing its lead experimental drug for use in common warts.

The drug, A-101, was tested in a 502-patient study called THWART-2 — patients enrolled had one to six warts before qualifying for the trial. Patients either self-administered A-101 topical solution or a vehicle twice a week over a two-month period. A higher proportion of patients on the drug (a potent hydrogen peroxide topical solution) saw their warts disappear at day 60, versus the vehicle (p<0.0001) — meeting the main goal of the study.  Each secondary endpoint also emerged in favor of A-101, the company said.

Charles Nichols, LSU School of Medicine

Could psy­che­delics tack­le the obe­si­ty cri­sis? A long­time re­searcher in the field says his lat­est mouse study sug­gests po­ten­tial

Psychedelics have experienced a renaissance in recent years amid a torrent of preclinical and clinical research suggesting it might provide a path to treat mood disorders conventional remedies have only scraped at. Now a preclinical trial from a young biotech suggests at least one psychedelic compound has effects beyond the mind, and — if you believe the still very, very early hype — could provide the first single remedy for some of the main complications of obesity.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Ac­celeron drops a de­vel­op­ment pro­gram as #2 drug fails to spark func­tion­al ben­e­fits in pa­tients with a rare neu­ro­mus­cu­lar ail­ment

Acceleron is scrapping a muscular dystrophy development program underway for its number 2 drug in the pipeline after pouring over some failed mid-stage secondary data.

Gone is the ACE-083 project in patients with facioscapulohumeral muscular dystrophy. Their drug hit the primary endpoint on building muscle but flopped on key secondaries for functional improvements in patients, which execs felt was vital to the drug’s success.