Es­pe­ri­on re­leas­es pos­i­tive PhII re­sults; Safe­ty warn­ings at In­dia, Chi­na man­u­fac­tur­ing sites spell trou­ble for glob­al sup­ply chain

→ Af­ter lin­ing up a new $200 mil­lion fund­ing deal for the pro­ject­ed com­mer­cial launch of be­mpe­doic acid next year, Es­pe­ri­on an­nounced to­day pos­i­tive re­sults from their Phase II ran­dom­ized, dou­ble-blind par­al­lel-group study of be­mpe­doic acid/eze­tim­ibe com­bi­na­tion tablet in com­par­i­son to eze­tim­ibe and place­bo. 179 pa­tients with hy­per­c­ho­les­terolemia and type II di­a­betes were en­rolled. The 12-week study met its pri­ma­ry and sec­ondary end­points.

The Phar­ma­ceu­ti­cal Jour­nal has con­duct­ed an analy­sis that shows the ma­jor­i­ty of warn­ings from Eu­ro­pean and US drug reg­u­la­tors are to man­u­fac­tur­ing sites in In­dia and Chi­na. The da­ta in­di­cate from ear­ly 2018 un­til Au­gust 2019 that the FDA’s of­fice of man­u­fac­tur­ing qual­i­ty pub­lished 75 warn­ing let­ters to phar­ma­ceu­ti­cal man­u­fac­tur­ers that vi­o­lat­ed its safe­ty and/or qual­i­ty stan­dards (49% of which could re­sult in en­force­ment ac­tion were sent to com­pa­nies in Chi­na or In­dia). Sim­i­lar­ly, the EMA is­sued 22 com­pli­ance no­tices, of which 14 (64%) were for man­u­fac­tur­ers in In­dia or Chi­na.

Cas­es such as the re­call­ing of thou­sands of batch­es of val­sar­tan-con­tain­ing prod­ucts that were con­t­a­m­i­nat­ed with po­ten­tial car­cino­gens in June 2018 from two fac­to­ries in Chi­na and the 2016 fac­to­ry ex­plo­sion in Chi­na re­sult­ing in se­vere short­ages of the API for piperacillin-tazobac­tam are rais­ing con­cerns of the glob­al sup­ply chain from ex­perts.

“Where I am con­cerned is the con­cen­tra­tion in pro­duc­tion of APIs — that is what should keep peo­ple awake at night,” says War­wick Smith, di­rec­tor-gen­er­al of the British Gener­ic Man­u­fac­tur­ers As­so­ci­a­tion. “If you on­ly have one or two man­u­fac­tur­ers for APIs for a crit­i­cal prod­uct, that is a re­al weak­ness in the sup­ply chain.”

A par­lia­men­tary re­port found that In­dia and Chi­na are among the top five providers of UK med­i­cines out­side the EU. A spokesper­son for the Med­i­cines and Health­care prod­ucts Reg­u­la­to­ry Agency (MHRA) said that it was not ow­ing to low­er stan­dards that these man­u­fac­tur­ers were re­ceiv­ing such high num­bers of no­tices, but be­cause of their larg­er mar­ket share and that there isn’t a sig­nif­i­cant dif­fer­ence in the num­ber of se­ri­ous non-com­pli­ance is­sues iden­ti­fied at UK site in com­par­i­son to non-EU coun­tries.

“Ac­cord­ing to da­ta pro­vid­ed by the MHRA, 5% of in­spec­tions of man­u­fac­tur­ers in In­dia found “crit­i­cal de­fi­cien­cies” in 2017 and 2018, com­pared with 4% of man­u­fac­tur­ers in the UK. How­ev­er, the da­ta al­so re­vealed that 6% of in­spec­tions of man­u­fac­tur­ers in In­dia re­sult­ed in the reg­u­la­tor is­su­ing a state­ment of non-com­pli­ance (SNC). This is com­pared with 0.8% of in­spec­tions of man­u­fac­tur­ers in the UK.”

→ Cam­bridge, Mass­a­chu­setts-based Har­bour Bio­Med is team­ing up with CRO Phar­ma­ceu­ti­cal Prod­uct De­vel­op­ment (PPD) to de­vel­op Har­bour’s ther­a­peu­tics in the fields of on­col­o­gy and im­munol­o­gy. The two com­pa­nies re­cent­ly com­plet­ed an Aus­tralian ethics com­mit­tee sub­mis­sion for HBM4003, an an­ti-can­cer im­munother­a­py to tar­get sol­id tu­mors — with a plan for Phase I tri­als in Aus­tralia and the US.

Bain-backed Spring­Works — which closed a se­ries B round in April, is now gun­ning for an IPO — has scored break­through ther­a­py sta­tus from the FDA for its ex­per­i­men­tal drug, nirogace­s­tat, which is a gam­ma-sec­re­tase in­hibitor un­der de­vel­op­ment to help pa­tients with pro­gres­sive, un­re­sectable, re­cur­rent or re­frac­to­ry desmoid tu­mors or deep fi­bro­mato­sis.

Soli­genix $SGNX has up­dat­ed the late-stage pro­gram for its ex­per­i­men­tal drug — SGX942 — for mu­cosi­tis, which is char­ac­ter­ized by dam­age done to the mu­cosa by an­ti­cancer ther­a­pies. On Wednes­day, the com­pa­ny said an in­de­pen­dent da­ta mon­i­tor­ing pan­el rec­om­mend­ed it in­crease en­roll­ment by 70 pa­tients to in­crease the to­tal to 260 sub­jects. The pan­el’s rec­om­men­da­tion in­di­cates that “a ben­e­fi­cial SGX942 ef­fect has been ob­served — how­ev­er, to main­tain the rig­or­ous as­sump­tion of 90% sta­tis­ti­cal pow­er for the pri­ma­ry ef­fi­ca­cy end­point, an in­crease was re­quired to take in­to ac­count any po­ten­tial vari­abil­i­ty and/or dis­tri­b­u­tion changes ob­served in the Phase 3 study pa­tient pop­u­la­tion that may have dif­fered from the ini­tial pro­to­col de­sign as­sump­tions,” Soli­genix said. Topline re­sults from the study are still ex­pect­ed in the first half of 2020, it added.

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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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.

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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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.