FDA press­es its cam­paign against rogue re­gen­er­a­tive med play­ers, cites Stem­Genex for il­le­gal sales

The FDA’s cam­paign against the “bad ac­tors” op­er­at­ing in re­gen­er­a­tive med­i­cine is far from com­plete.

The reg­u­la­tor on Tues­day ad­mon­ished San Diego-based Stem­Genex Bi­o­log­ic Lab­o­ra­to­ries LLC for mar­ket­ing an un­ap­proved stem cell prod­uct and point­ed out sig­nif­i­cant vi­o­la­tions that could cor­rupt the man­u­fac­tur­ing process, po­ten­tial­ly cul­mi­nat­ing in mi­cro­bial con­t­a­m­i­na­tion that put pa­tients at risk.

The crack­down on clin­ics sell­ing re­gen­er­a­tive med­i­cines for a host of un­ap­proved in­di­ca­tions con­tin­ues to gath­er steam, as the agency makes good on its 2017 promise to en­sure un­scrupu­lous play­ers mar­ket­ing un­proven and of­ten dan­ger­ous prod­ucts are held to ac­count.

The field en­ticed a grow­ing list of com­pa­nies that have “op­por­tunis­ti­cal­ly seized on the clin­i­cal po­ten­tial of re­gen­er­a­tive med­i­cine” to make du­bi­ous claims to pa­tients that of­ten suf­fer from in­cur­able, de­bil­i­tat­ing dis­eases, the agency un­der­scored in a state­ment in Au­gust 2017.


In Stem­Genex’s case, the FDA found the com­pa­ny was pro­cess­ing body fat in­to stro­mal vas­cu­lar frac­tion (SVF) — a cel­lu­lar prod­uct — and “il­le­gal­ly” mar­ket­ing it for se­ri­ous, of­ten life-threat­en­ing dis­eases, in­clud­ing di­a­betes, fi­bromyal­gia, spinal cord in­jury, chron­ic ob­struc­tive pul­monary dis­ease and mus­cu­lar dy­s­tro­phy. And they ad­min­is­ter it by IV, in­hala­tion and di­rect­ly in­to the spinal canal.

This prod­uct, which in­volves more than the min­i­mal ma­nip­u­la­tion of a pa­tient’s adi­pose tis­sue, is reg­u­lat­ed as both a drug and bi­o­log­i­cal prod­uct, but the agency said that no such li­cens­es or ap­provals have been sanc­tioned or ap­plied for.

On the com­pa­ny’s web­site, its pro­pri­etors claim:

Stem­Genex Stem Cell Re­search Cen­tre and af­fil­i­ates do not claim that treat­ment us­ing au­tol­o­gous stem cells are a cure for any con­di­tion, dis­ease, or in­jury. Adi­pose-de­rived stem cell ther­a­py is not a part of FDA ap­proved stem cell ther­a­pies and is not a con­sid­ered a cure for any med­ical con­di­tion. All state­ments and opin­ions pro­vid­ed on this web­site are for ed­u­ca­tion­al and in­for­ma­tion­al pur­pos­es on­ly and we do not di­ag­nose or give med­ical ad­vise via this web­site.

As part of its warn­ing let­ter sent to Stem­Genex on Oc­to­ber 31, FDA al­so list­ed a num­ber of de­vi­a­tions from cur­rent good man­u­fac­tur­ing prac­tice re­quire­ments in the man­u­fac­ture of the SVF prod­uct, in­clud­ing un­val­i­dat­ed man­u­fac­tur­ing process­es and an un­con­trolled en­vi­ron­ment.

The warn­ing let­ter is the lat­est in a slate of warn­ings is­sued by the reg­u­la­tor since last year. Last Jan­u­ary, the FDA slapped a sim­i­lar warn­ing on New Jer­sey-based Amer­i­can CryoStem Corp, in ad­di­tion to warn­ings is­sued to or­ga­ni­za­tions in Flori­da and Cal­i­for­nia in 2017 to ad­dress such prod­ucts be­ing mar­ket­ed.

John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.
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The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

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UP­DAT­ED: Chica­go biotech ar­gues blue­bird, Third Rock 'killed' its ri­val, pi­o­neer­ing tha­lassemia gene ther­a­py in law­suit

Blue­bird bio $BLUE chief Nick Leschly court­ed con­tro­ver­sy last week when he re­vealed the com­pa­ny’s be­ta tha­lassemia treat­ment will car­ry a jaw-drop­ping $1.8 mil­lion price tag over a 5-year pe­ri­od in Eu­rope — mak­ing it the plan­et’s sec­ond most ex­pen­sive ther­a­py be­hind No­var­tis’ $NVS fresh­ly ap­proved spinal mus­cu­lar at­ro­phy ther­a­py, Zol­gens­ma, at $2.1 mil­lion. A Chica­go biotech, mean­while, has been fum­ing at the side­lines. In a law­suit filed ear­li­er this month, Er­rant Gene Ther­a­peu­tics al­leged that blue­bird and ven­ture cap­i­tal group Third Rock un­law­ful­ly prised a vi­ral vec­tor, de­vel­oped in part­ner­ship with the Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter (MSK), from its grasp, and thwart­ed the de­vel­op­ment of its sem­i­nal gene ther­a­py.

John Chiminski, Catalent CEO - File Photo

'It's a growth play': Catal­ent ac­quires Bris­tol-My­er­s' Eu­ro­pean launch pad, ex­pand­ing glob­al CD­MO ops

Catalent is staying on the growth track.

Just two months after committing $1.2 billion to pick up Paragon and take a deep dive into the sizzling hot gene therapy manufacturing sector, the CDMO is bouncing right back with a deal to buy out Bristol-Myers’ central launchpad for new therapies in Europe, acquiring a complex in Anagni, Italy, southwest of Rome, that will significantly expand its capacity on the continent.

There are no terms being offered, but this is no small deal. The Anagni campus employs some 700 staffers, and Catalent is planning to go right in — once the deal closes late this year — with a blueprint to build up the operations further as they expand on oral solid, biologics, and sterile product manufacturing and packaging.

This is an uncommon deal, Catalent CEO John Chiminski tells me. But it offers a shortcut for rapid growth that cuts years out of developing a green fields project. That’s time Catalent doesn’t have as the industry undergoes unprecedented expansion around the world.

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Arc­turus ex­pands col­lab­o­ra­tion, adding $30M cash; Ku­ra shoots for $100M raise

→  Rare dis­ease play­er Ul­tragenyx $RARE is ex­pand­ing its al­liance with Arc­turus $ARCT, pay­ing $24 mil­lion for eq­ui­ty and an­oth­er $6 mil­lion in an up­front as the two part­ners ex­pand their col­lab­o­ra­tion to in­clude up to 12 tar­gets. “This ex­pand­ed col­lab­o­ra­tion fur­ther so­lid­i­fies our mR­NA plat­form by adding ad­di­tion­al tar­gets and ex­pand­ing our abil­i­ty to po­ten­tial­ly treat more dis­eases,” said Emil Kakkis, the CEO at Ul­tragenyx. “We are pleased with the progress of our on­go­ing col­lab­o­ra­tion. Our most ad­vanced mR­NA pro­gram, UX053 for the treat­ment of Glyco­gen Stor­age Dis­ease Type III, is ex­pect­ed to move in­to the clin­ic next year, and we look for­ward to fur­ther build­ing up­on the ini­tial suc­cess of this part­ner­ship.”

Neil Woodford. Woodford Investment Management via YouTube

Wood­ford braces po­lit­i­cal storm as UK fi­nan­cial reg­u­la­tors scru­ti­nize fund sus­pen­sion

The shock of Neil Wood­ford’s de­ci­sion to block with­drawals for his flag­ship fund is still rip­pling through the rest of his port­fo­lio — and be­yond. Un­der po­lit­i­cal pres­sure, UK fi­nan­cial reg­u­la­tors are now tak­ing a hard look while in­vestors con­tin­ue to flee.

In a re­sponse let­ter to an MP, the Fi­nan­cial Con­duct Au­thor­i­ty re­vealed that it’s opened an in­ves­ti­ga­tion in­to the sus­pen­sion fol­low­ing months of en­gage­ment with Link Fund So­lu­tions, which tech­ni­cal­ly del­e­gat­ed Wood­ford’s firm to man­age its funds.

Gilead baits new al­liance with $45M up­front, div­ing in­to the busy pro­tein degra­da­tion field

Gilead is jump­ing on board the pro­tein degra­da­tion band­wag­on. And they’re turn­ing to a low-pro­file Third Rock start­up for the ex­per­tise. But if you were look­ing for a trans­for­ma­tion­al deal to kick up fresh en­thu­si­asm for Gilead, you’ll have to re­main pa­tient.

This one will have a long way to go be­fore they get in­to the clin­ic.

The big biotech said Wednes­day morn­ing that it is pay­ing $45 mil­lion up­front and re­serv­ing a whop­ping $2.3 bil­lion in biotech bucks if San Fran­cis­co-based Nurix can point the way to new can­cer ther­a­pies, as well as drugs for oth­er, un­spec­i­fied dis­eases.

A new num­ber 1 drug? Keytru­da tapped to top the 10 biggest block­busters on the world stage by 2024

Analysts may be fretting about Keytruda’s longterm prospects as a host of rival therapies elbow their way to the market. But the folks at Evaluate Pharma are confident that last year’s $7 billion earner is headed for glory, tapping it to beat out the current #1 therapy Humira as AbbVie watches that franchise swoon over the next 5 years.

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In­vestor day prep at Mer­ck in­cludes a new strat­e­gy to pick up the pace on M&A — re­port

Mer­ck’s re­cent deals to buy up two bolt-on biotechs — Ti­los and Pelo­ton — weren’t an aber­ra­tion. In­stead, both ac­qui­si­tions mark a new strat­e­gy to beef up its dom­i­nant can­cer drug op­er­a­tions cen­tered on Keytru­da while look­ing to ad­dress grow­ing con­cerns that too many of its eggs are in the one I/O bas­ket for their PD-1 pro­gram. And Mer­ck is go­ing af­ter more small- and mid-sized buy­outs to calm those fears.