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.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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Ken Frazier, AP Images

Why Mer­ck wait­ed, and what they now bring to the Covid-19 fight

Nicholas Kartsonis had been running clinical infectious disease research at Merck for almost 2 years when, in mid-January, he got a new assignment: Searching the Pharma giant’s vast libraries for something that could treat the novel coronavirus.

The outbreak was barely two weeks old when Kartsonis and a few dozen others got to work, first in small teams and then in a larger task force that sucked in more and more parts of the sprawling company as Covid-19 infected more and more of the globe. By late February, the group began formally searching for vaccine and antiviral candidates to license. Still, while other companies jumped out to announce their programs and, eventually and sometimes controversially, early glimpses at human data, Merck remained silent. They made only a brief announcement about a data collection partnership in April and mentioned vaguely a vaccine and antiviral search in their April 28 earnings call.

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Mark Genovese (Stanford via Twitter)

Gilead woos fil­go­tinib clin­i­cal in­ves­ti­ga­tor from Stan­ford to lead the charge on NASH, in­flam­ma­to­ry dis­eases

With an FDA OK for the use of filgotinib in rheumatoid arthritis expected to drop any day now, Gilead has recruited a new leader from academia to lead its foray into inflammatory diseases.

Mark Genovese — a longtime Stanford professor and most recently the clinical chief in the division of immunology and rheumatology — was the principal investigator in FINCH 2, one of three studies that supported Gilead’s NDA filing. In his new role as SVP, inflammation, he will oversee the clinical development of the entire portfolio.

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Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

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Federico Mingozzi (Spark)

Spark touts an­i­mal da­ta for a so­lu­tion to AAV gene ther­a­py's an­ti­body prob­lem

Among all the limitations of using an adeno-associated virus as a vector to deliver a gene — still the most established modality in gene therapy given years of trial and error and finally success — the presence of neutralizing antibodies, whether pre-existing or induced, looms large.

“When I think about the immune responses in AAV, I try to sort of layer them,” Federico Mingozzi, the CSO at Spark Therapeutics, told Endpoints News. “The antibody is the first layer. It’s the first block that you find when you’re trying to do gene transfer.”

Stephen Isaacs, Aduro president and CEO (Aduro)

Once a high fly­er, a stag­ger­ing Aduro is auc­tion­ing off most of the pipeline as CEO Stephen Isaacs hands off the shell to new own­ers

After a drumbeat of failure, setbacks and reorganizations over the last few years, Aduro CEO Stephen Isaacs is handing over his largely gutted-out shell of a public company to another biotech company and putting up some questionable assets in a going-out-of-business sale.

Isaacs —who forged a string of high-profile Big Pharma deals along the way — has wrapped a 13-year run at the biotech with one program for kidney disease going to the new owners at Chinook Therapeutics. A host of once-heralded assets like their STING agonist program partnered with Novartis (which dumped their work on ADU-S100 after looking over weak clinical results), the Lilly-allied cGAS-STING inhibitor program and the anti-CD27 program out-licensed to Merck will all be posted for auction under a strategic review process.

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Hill­house re­casts spot­light on Chi­na's biotech scene with $160M round for Shang­hai-based an­ti­body mak­er

Almost two years after first buying into Genor Biopharma’s pipeline of cancer and autoimmune therapies, Hillhouse Capital has led a $160 million cash injection to push the late-stage assets over the finish line while continuing to fund both internal R&D and dealmaking.

The Series B has landed right around the time Genor would have listed on the Hong Kong stock exchange, according to plans reported by Bloomberg late last year. Insiders had said that the company was looking to raise about $200 million.

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No­var­tis chips in $10M for IPO-bound part­ner Pli­ant; Tenax shares soar on heart drug da­ta

Novartis is coming in with $10 million to help support the looming IPO of a partner. Pliant Therapeutics posted a new filing with the SEC showing that Novartis is buying the shares at $15, the mid-point of the range. It’s adding several million shares to the offering, bringing the total to around $135 million. Biotech companies have been enjoying quite a run on virtual Wall Street, with investors boosting new offerings to some big hauls.