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.

A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

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David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

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While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

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Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.

Jeff Kindler's Cen­trex­ion re­news bid to make pub­lic de­but

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.

Zachary Hornby. Boundless

'A fourth rev­o­lu­tion in can­cer ther­a­pies': ARCH-backed Bound­less Bio flash­es big check, makes big­ger promis­es in de­but

It was the cellular equivalent of opening your car door and finding an active, roaring engine in the driver seat.

Scientists learned strands of DNA could occasionally appear outside of its traditional home in the nucleus in the 1970s, when they appeared as little, innocuous circles on microscopes; inexplicable but apparently innate. But not until UC San Diego’s Paul Mischel published his first study in Science in 2014 did researchers realize these circles were not only active but potentially overactive and driving some cancer tumors’ superhuman growth.

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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Mer­ck helps bankroll new part­ner Themis' game plan to fin­ish the chikun­gun­ya race and be­gin on­colyt­ic virus quest

As Themis gears up for a Phase III trial of its chikungunya vaccine, the Vienna-based biotech has closed out €40 million ($44 million) to foot the clinical and manufacturing bills.

Its heavyweight partners at Merck — which signed a pact around a mysterious “blockbuster indication” last month — jumped into the Series D, led by new investors Farallon Capital and Hadean Ventures. Adjuvant Capital also joined, as did current investors Global Health Investment Fund, aws Gruenderfonds, Omnes Capital, Ventech and Wellington Partners Life Sciences.