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.

UP­DAT­ED: Roche bags 'break­through' an­ti-fi­bro­sis drug in $1.4B biotech buy­out deal

Roche is snapping up a “breakthrough” anti-fibrotic drug in a $1.4 billion buyout.

The pharma giant announced Friday that it is acquiring Promedior, primarily to get its hands on PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein that has nailed down mid-stage clinical data on idiopathic pulmonary fibrosis and demonstrating its potential for a range of fibrotic conditions.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,600+ biopharma pros reading Endpoints daily — and it's free.

(Image: Associated Press)

Amarin emerges from an ex­pert pan­el re­view with a clear en­dorse­ment for Vas­cepa and high odds of suc­cess when the FDA weighs in for­mal­ly

Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,600+ biopharma pros reading Endpoints daily — and it's free.

No­var­tis spin­out’s first an­ti-ag­ing PhI­II is a flop, so now they’ll turn to Parkin­son’s chal­lenge as shares wilt

Novartis spinout resTORbio is grappling with the collapse of its lead clinical program this morning — an anti-aging R&D failure that will badly damage their rep in the field.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,600+ biopharma pros reading Endpoints daily — and it's free.

(Image: Associated Press)

No­var­tis scores its lat­est FDA OK — this time for a new sick­le cell dis­ease drug picked up in a $665M deal

Novartis’ decision to buy Oklahoma-based biotech Selexys 3 years ago for up to $665 million has paid off with an FDA approval today.

Blessed with the FDA’s breakthrough drug designation for a speedy review, the pharma giant has pinned down an approval for crizanlizumab, a new therapy designed to reduce the frequency of painful incidents of vaso-occlusive crises among sickle cell disease patients 16 or older.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 65,600+ biopharma pros reading Endpoints daily — and it's free.

As­traZeneca gains EU nod for di­a­betes triple; Am­gen and Duke launch re­al-world PC­SK9 ob­ser­va­tion­al study

→ Weeks after winning EU approval to start marketing dapagliflozin as Forxiga, AstraZeneca has racked up another OK for a triplet combo involving the SGLT2 diabetes drug. Named Qtrilmet, the pill combines Forxiga with the DPP-4 inhibitor Onglyza (saxagliptin) and the bedrock drug metformin in a modified-release format. That 3-in-1 approach proved superior in reducing average blood glucose levels to a number of other dual combinations across 5 Phase III trials, including Forxiga plus metformin, Onglyza with metformin, or glimepiride with metformin.

Five drugs, in­clud­ing two No­var­tis ther­a­pies, win EMA en­dorse­ment

As is custom, an EMA panel on Friday issued its weekly recommendations on marketing applications submitted by drug developers. This week, the agency backed the use of five new therapies — including two Novartis drugs — but issued no negative reviews.

Novartis’ S1P drug for relapsing forms of multiple sclerosis (MS) drug, Mayzent (known chemically as siponimod), which was approved by the FDA in March — has been given the nod by the EMA. The Swiss drugmaker already sells its other MS drug, Gilenya, in both regions.

Atom­wise's X-37 spin­out gets $14.5 mil­lion to launch AI dis­cov­ery ef­forts

The folks behind Atomwise’s spinout X-37 like to think in cosmological metaphors, and you can think of their AI drug development model as probes sent into space from a central station. That station just got $14.5 million in Series A funding from DCVC Bio, Alpha Intelligence Capital and Hemi Ventures to back those missions.

X-37 uses Atomwise’s AI platform to identify drug targets and – unlike the parent company, which largely sticks to computers  – bring those into a wet lab and preclinical testing.  In addition to AI professionals, it’s led in by part by drug developers from Velocity Pharmaceutical Development.

Ab­bott Lab­o­ra­to­ries CEO Miles White pass­es ba­ton down to suc­ces­sor; Lon­za CEO Marc Funk hits the ex­it

→ Abbott Laboratories has named a successor to CEO Miles White after he announced that he was stepping down in March after 21 years of service. Robert Ford, the company’s COO and president, will take the helm. Ford is known for his work in the $25 billion merger between St. Jude Medical into Abbott in January 2017. White will remain with the company as executive chairman of the board. 

→ After snapping up Novartis’ Swiss facility, Novartis Center of Excellence, in July, Lonza has announced that their CEO, Marc Funk, is hitting the exit for “personal reasons.” Funk has been the CEO of the company for less than a year — brought onto the company back in March. In the meantime, chairman Albert Baehny will serve as interim CEO. 

UCB adds on more pos­i­tive PhI­II da­ta for IL-17A/17F in­hibitor bimek­izum­ab, clear­ing a path to the FDA

A month after posting positive top-line data from their first Phase III trial of the IL-17A/17F inhibitor bimekizumab, Belgium’s UCB says they’ve added more upbeat results from their second late-stage test in moderate-to-severe plaque psoriasis.

That leaves the company on track for regulatory submissions in the middle of next year, says CMO Iris Loew-Friedrich.
Their drug beat out a placebo on the co-primaries — a 90% improvement in PASI 90 (the Psoriasis Area and Severity Index) and Investigator Global Assessment (IGA) response of clear or almost clear (IGA 0/1) at week 16, compared to placebo. Investigators also boasted of hitting some key secondaries.
UCB is angling to enter an increasingly crowded market space.
In their first of 3 Phase III studies for bimekizumab, researchers touted top-line wins on statistically significant results on clearing plaque psoriasis, including a victory over J&J’s IL-23 contender Stelara on key endpoints. The drug targets both IL-17A and IL-17F, a modification on the IL-17A strategy laid out for Taltz (Eli Lilly) and Cosentyx (Novartis). And the new group also includes J&J’s Tremfya and AbbVie’s Skyrizi.

Social image: UCB