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.

Andre Kalil, AP Images

A 9/11-era Om­a­ha fa­cil­i­ty, an old Ebo­la drug, and the ubiq­ui­tous Dr. Fau­ci: In­side the first US nov­el coro­n­avirus tri­al

The first 11 coronavirus patients who arrived in Omaha last week, airlifted across the globe after two weeks quarantined on a cruise ship, showed only minor symptoms or none at all. And then one of them — or one of the couple of Americans who arrived later — got worse. He developed pneumonia, a life-threatening complication for coronavirus patients.

In a biocontainment room at the University of Nebraska Medical Center on Friday, doctors infused him with an experimental Gilead drug once developed for Ebola, called remdesivir. Or they gave him a placebo. For the first time in the US, neither he nor the doctors knew.

The first US novel coronavirus trial was underway and with it, a mad dash for an answer. Sponsored by the NIH, the study marked a critical point in the epidemic. Since the start of the outbreak, the agency had helped lead a global effort to contain the virus. Now, as it spread worldwide and the CDC issued warnings the US could see a major internal outbreak, they were looking at home.

“We don’t have too much time,” Andre Kalil, the trial’s lead investigator, told Endpoints News. “Everything’s moving really fast.”

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Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Take­da swoops in to buy lit­tle biotech part­ner and its celi­ac drug poised to 'change stan­dard of care'

Having spent three years carefully grooming PvP Biologics and its drug for celiac disease, Takeda is happy enough with the proof-of-concept data to buy it all.

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A name emerges out of the Gilead M&A ru­mor mill, and it’s a can­cer biotech

After months of questions and speculation about when and if Gilead will make a major acquisitions, a name has emerged.

The California-based drugmaker has approached Forty Seven Inc, a cancer biotech, with a takeover offer, Bloomberg News reports. With Forty Seven’s market cap at $2.3 billion, an acquisition would likely be Gilead’s largest since they acquired Kite Pharma for $11.9 billion in 2017.

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Biogen head of R&D Al Sandrock, Sangamo CEO Sandy Macrae

UP­DAT­ED: Bio­gen makes an­oth­er bold Alzheimer’s bet, drop­ping $350M up­front to part­ner with genome-edit­ing fo­cused Sang­amo

While the fate of Biogen’s resurrected Alzheimer’s drug aducanumab remains uncertain, the Cambridge, MA-based drugmaker is joining forces with genome editing company Sangamo Therapeutics to develop therapies for neurological conditions.

Sangamo is set to receive a meaty $350 million upfront in cash and stock and is eligible to receive up to $2.37 billion in milestone payments, in addition to royalties. In return, Biogen gets the rights to two Sangamo preclinical compounds: ST-501 (for use in tauopathies including Alzheimer’s disease) and ST-502 (for synucleinopathies including Parkinson’s disease).

“The partnership represents a lower-cost way to expand its work in neurologic disease,” Credit Suisse’s Evan Seigerman said in a note, referring to Biogen.

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Spark los­es an­oth­er top ex­ec in the wake of $4.3B takeover by Roche — re­port

Days after bidding farewell to co-founder Kathy High, Spark Therapeutics — now operating under Roche — has one more opening on its C-suite.

Kathy Reape

Kathy Reape, who joined the Philadelphia-based biotech in 2016 as head of clinical R&D and became chief medical officer in 2018, is reportedly set to leave.

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'The head­lines are the head­lines, but': Bio­Marin talks up po­ten­tial sav­ings as he­mo­phil­ia gene ther­a­py launch looms

BioMarin execs are still staying tight-lipped about their pricing plans for what is poised to be the world’s first hemophilia gene therapy. But as the company enters the final regulatory stretch and approaches a potential launch this summer, they are also dropping more hints to get investors ready.

First thing to know: They really, really don’t expect an advisory committee to be convened for valrox, which is under priority review, to pop up before its PDUFA date on August 21.

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Vlad Coric (Photo Credit: Andrew Venditti)

Bio­haven scores CGRP OK for acute mi­graine — can the com­mer­cial team catch up with Al­ler­gan on its de­but?

Seven years after spinning out of Yale, Biohaven has entered the ranks of commercial-stage biotechs.

The FDA handed down an OK for its CGRP drug, rimegepant, as an acute treatment. Dubbed Nurtec, the orally dissolving pill will join Allergan’s (soon to be AbbVie’s) Ubrelvy and Lilly’s Reyvow on the market amid a new wave of migraine therapies reshaping the disease space.

In a pivotal Phase III trial, Nurtec hit the co-primary endpoints on pain freedom and freedom from most bothersome symptoms at two hours post dose, proving superior to placebo.

Mi­cro­bio­me Q&A: New study maps the vagi­na's 'op­ti­mal mi­cro­bio­ta' — and its im­pli­ca­tions for bio­phar­ma

The widely-held notion that the “optimal” vaginal microbiota is dominated by one strain of lactic-acid producing bacteria has now been challenged in a new paper, published in Nature Communications on Wednesday, which used advanced gene sequencing methods to map out the most comprehensive gene catalog of the human vaginal microbiome.

Things have changed in the more than 50 years since the concept of vaginal microbiota transplants was proposed and subsequently tainted by a Texas-based gynecologist who transplanted the vaginal fluid of women who had bacterial vaginosis into healthy females, suspecting he had isolated the bacteria responsible for the condition.

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