Ben-Gu­ri­on Uni­ver­si­ty seek­ing part­ner to re­pur­pose old lym­phoma drug to treat ALS

Re­searchers at the Ben-Gu­ri­on Uni­ver­si­ty of the Negev are seek­ing col­lab­o­ra­tors who can help them teach an old lym­phoma drug a new trick- to treat ALS.

Rachel Lichen­stein, a re­searcher at Ben-Gu­ri­on in Is­rael had been study­ing the role of sug­ar mol­e­cules in can­cer when a stu­dent drew her at­ten­tion to a lab in a near­by hos­pi­tal that hap­pened to have 20 tis­sue sam­ples from ALS pa­tients. So Lichen­stein fig­ured she might as well check them out.

“I came by ALS by co­in­ci­dence,” she ex­plained to End­points News, “and Bin­go! We found some­thing.”

What she found was a unique sug­ar mol­e­cule in the sam­ples that in­creas­es an­ti­body’s abil­i­ty to bind to im­mune cells by about 100 times. In the late stages of ALS, these an­ti­bod­ies guide im­mune cells to at­tack healthy mo­tor neu­rons.

So Lichen­stein set out to pre­vent the an­ti­body from bind­ing to the im­mune cells in the first place.

She start­ed with Rit­ux­imab, a mon­o­clon­al an­ti­body tar­get­ing CD20, that is al­ready ap­proved to treat some lym­phomas. Splic­ing the mol­e­cule to re­move the por­tion de­signed to kill can­cer, leav­ing on­ly the sug­ar that binds to the an­ti­body, she cre­at­ed a new mol­e­cule she be­lieves will block im­mune cells from bind­ing to the an­ti­body, and thus pre­vent the cells from at­tack­ing mo­tor neu­rons.

So far, she says a sin­gle in­jec­tion of the drug be­fore the on­set of the dis­ease, ex­tend­ed life ex­pectan­cy in a mouse mod­el of ALS about three weeks. The mice al­so showed signs that their brain cells were bet­ter able to clear de­bris af­ter the in­jec­tion — a process that is dis­rupt­ed in oth­er neu­rode­gen­er­a­tive dis­eases as well. Of course, pro­long­ing the life of a mouse a few weeks is a long way from mean­ing­ful­ly ex­tend­ing and im­prov­ing the lives of hu­mans suf­fer­ing from ALS, or any oth­er dis­ease.

Still, the team is op­ti­mistic. “Since the drug is al­ready ap­proved, we be­lieve that we will on­ly need lim­it­ed pre­clin­i­cal test­ing to reach the clin­i­cal phase ear­li­er than oth­er ini­tia­tives,” said Lichen­stein in a press re­lease.

When asked if she had pur­sued a col­lab­o­ra­tion with the drug’s man­u­fac­tur­er, Lichen­stein told me “It is in our plan to con­tact the man­u­fac­tur­er of Rit­ux­imab to ask for part­ner­ship.”

Like most neu­rode­gen­er­a­tive dis­eases, pa­tient’s op­tions for treat­ment are lim­it­ed and un­der­whelm­ing. There are so few op­tions that when FDA reg­u­la­tors heard about a drug ap­proved in Japan and Ko­rea, edar­avone, they reached out to the man­u­fac­tur­er, Mit­subishi Tan­abe, and re­quest­ed that the com­pa­ny file for ap­proval in the Unit­ed States. It was ap­proved about a month ago. Be­fore that, there was on­ly one oth­er drug for ALS, an antiglu­ta­mate called Rilu­zole, de­vel­oped by Sanofi and ap­proved in 1995. Both slow the pro­gres­sion of the dis­ease slight­ly, giv­ing pa­tients a pre­cious few ex­tra months to a cou­ple years of mo­tor con­trol. How­ev­er, edavarone is ad­min­is­tered in­tra­venous­ly, which has raised con­cerns. It al­so may come with an un­af­ford­able price tag for many af­flict­ed.

Lichen­stein says the main ob­sta­cles are, of course, time and mon­ey.

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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How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

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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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.”

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.

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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