Sci­en­tists hold out hope for re­gen­er­a­tive in­fu­sion via brain im­plant — de­spite fail­ing its first test in Parkin­son's

A re­gen­er­a­tive ap­proach to treat­ing Parkin­son’s dis­ease — fea­tur­ing an im­plant to pump a pro­tein di­rect­ly in­to pa­tients’ brains — floun­dered on its first clin­i­cal test, but that hasn’t stopped re­searchers in the UK from hold­ing out hope that it can even­tu­al­ly work by restor­ing dam­aged brain cells.

Cham­pi­oned by Parkin­son’s UK, the treat­ment works by boost­ing the lev­els of GDNF, or glial cell line de­rived neu­rotroph­ic fac­tor, a nat­u­ral­ly oc­cur­ring pro­tein thought to pro­tect cells and help them re­cov­er or re­grow. To re­ceive the pro­tein — which is too large to pen­e­trate the blood-brain bar­ri­er — pa­tients first have to un­der­go a ro­bot-as­sist­ed surgery to have four tubes placed in­to their brains, then get hooked to a ma­chine that in­fuse GDNF to pre­cise lo­ca­tions of the brain via a port on the side of the head.

Af­ter con­firm­ing safe­ty with six pa­tients, sci­en­tists re­cruit­ed 35 more pa­tients to see if in­fus­ing GDNF this way led to im­prove­ments in Parkin­son’s symp­toms, from mo­tor move­ments to dai­ly ac­tiv­i­ties.

The short an­swer is no: The “en­cour­ag­ing signs of im­prove­ments” among pa­tients in the drug arm did not con­sti­tute a sig­nif­i­cant dif­fer­ence from the place­bo group.

Alan Whone

But prin­ci­pal in­ves­ti­ga­tor Alan Whone was quick to of­fer po­ten­tial ex­pla­na­tions, name­ly the short dou­ble-blind tri­al time (nine months), the rel­a­tive­ly low dosage of GDNF, and the stage that pa­tients were al­ready at by the time they en­rolled.

His op­ti­mism is backed up by some ev­i­dence that GDNF had promis­ing ef­fects on brain cells al­ready rav­aged by the dis­ease — show­ing a 100% im­prove­ment in a key area of the brain af­fect­ed in the con­di­tion com­pared to place­bo, which didn’t in­duce any change.

“The spa­tial and rel­a­tive mag­ni­tude of the im­prove­ment in the brain scans is be­yond any­thing seen pre­vi­ous­ly in tri­als of sur­gi­cal­ly de­liv­ered growth-fac­tor treat­ments for Parkin­son’s,” Whone said in a state­ment. “This rep­re­sents some of the most com­pelling ev­i­dence yet that we may have a means to pos­si­bly reawak­en and re­store the dopamine brain cells that are grad­u­al­ly de­stroyed in Parkin­son’s.”

Fur­ther­more, a nine-month fol­low-up pe­ri­od dur­ing which the place­bo group al­so switched to GNDF saw all par­tic­i­pants demon­strat­ing “mod­er­ate to large im­prove­ment in symp­toms” com­pared to their own base­line scores, bol­ster­ing the hope that the ef­fects on symp­toms were just lag­ging be­hind im­prove­ment in brain cells.

Erich Mohr

All of this spells “ex­cit­ing signs of promise” for de­vice man­u­fac­tur­er Ren­ishaw as well as Med­Ge­n­e­sis, the biotech de­vel­op­ing GDNF and div­ing in­to an in­creas­ing­ly crowd­ed field with plen­ty of small­er play­ers tied up with big part­ners, tout­ing new tech­nolo­gies like pro­tein degra­da­tion to gene ther­a­py.

“We be­lieve this ex­per­i­men­tal Parkin­son’s Dis­ease Com­pos­ite Re­sponse (PD­CORE) may bet­ter cap­ture the full ef­fects of GDNF and we’re work­ing to get it sci­en­tif­i­cal­ly val­i­dat­ed so that it can be used in fu­ture tri­als,” CEO Erich Mohr said.

GDNF de­vice. Cred­it: Mint­Mo­tion for Pas­sion­ate Pro­duc­tions

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Hal Barron and Rick Klausner (GSK, Lyell)

Ex­clu­sive: GSK’s Hal Bar­ron al­lies with Rick Klaus­ner’s $600M cell ther­a­py start­up, look­ing to break new ground blitz­ing sol­id tu­mors

LONDON — Chances are, you’ve heard little or nothing about Rick Klausner’s startup Lyell. But that ends now.

Klausner, the former head of the National Cancer Institute, former executive director for global health at the Gates Foundation, co-founder at Juno and one of the leaders in the booming cell therapy field, has brought together one of the most prominent teams of scientists tackling cell therapy 2.0 — highlighted by a quest to bridge a daunting tech gap that separates some profound advances in blood cancers with solid tumors. And today he’s officially adding Hal Barron and GlaxoSmithKline as a major league collaborator which is pitching in a large portion of the $600 million he’s raised in the past year to make that vision a reality.

“We’ve being staying stealth,” Klausner tells me, then adding with a chuckle: “and going back to stealth after this.”

“Cell therapy has a lot of challenges,” notes Barron, the R&D chief at GSK, ticking off the resistance put up by solid tumors to cell therapies, the vein-to-vein time involved in taking immune cells out of patients, engineering them to attack cancer cells, and getting them back in, and more. “Over the years Rick and I talked about how it would be wonderful to take that on as a mission.”

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First place fin­ish: Eli Lil­ly just moved to fran­chise leader with their sec­ond mi­graine drug OK in 1 year

In a rare twist for Eli Lilly’s historically slow-moving R&D group, the pharma giant has seized bragging rights to a first-in-class new drug approval. And all signs point to an aggressive marketing followup as they look to outclass some major franchise rivals hobbled by internal dissension.

The FDA came through with an OK for lasmiditan on Friday evening, branding it as Reyvow and lining it up — once a substance classification comes through from the DEA — for a major market release. The oral drug binds to 5-HT1F receptors and is designed to stop an acute migraine after it starts. That makes it a complementary therapy to their CGRP drug Emgality, which has a statistically significant impact on preventing attacks.

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Allogene HQ Open House on September 17, 2019 in South San Francisco. (Jeff Rumans, Endpoints News)

The next 10 years: Where is biotech head­ed?

The last 10 years have seen a revolution in drug development. Timelines have shortened, particularly in oncology. Regulators have opened up. Investment has skyrocketed. China became a player. Biotechs have multiplied as gene and cell therapy has exploded — offering major new advances in the way diseases are treated, and sometimes cured.

So where are we headed from here? I journeyed out to San Francisco in September to discuss the answer to that question at Allogene’s open house. If the last 10 years have been an eye-opener, what does the next decade hold in store?

Patrick Mahaffy, Getty Images

Court green-lights Clo­vis case af­ter de­tail­ing ev­i­dence the board ‘ig­nored red flags’ on false safe­ty and ef­fi­ca­cy da­ta

Clovis investors have cleared a major hurdle in their long-running case against the board of directors, with a Delaware court making a rare finding that they had a strong enough case against the board to proceed with the action.

In a detailed ruling at the beginning of the month that’s been getting careful scrutiny at firms specializing in biotech and corporate governance, the Delaware Court of Chancery found that the attorneys for the investors had made a careful case that the board — a collection of experts that includes high-profile biotech entrepreneurs, a Harvard professor and well-known investigator as well as Clovis CEO Patrick Mahaffy — repeatedly ignored obvious warnings that Mahaffy’s executive crew was touting inflated, unconfirmed data for their big drug Roci. Serious safety issues were also reportedly overlooked while the company continued a fundraising campaign that brought in more than a half-billion dollars. And that leaves the board open to claims related to their role in the fiasco.

The bottom line:

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Bill Gates backs Gink­go Biowork­s' $350M raise to fu­el the buzzy syn­thet­ic bi­ol­o­gy 'rev­o­lu­tion'

If you want to understand Ginkgo Bioworks, the name should suffice: Bioworks, a spin off “ironworks,” that old industrial linchpin devoted to leveraging scale as a wellspring for vast new industries capable of remaking society. Ginkgo wants to be the ironworks for the revolution it’s heralded with as much fanfare as they can, playing off of one of the buzziest technologies in biotech.

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UCB bags a ri­val to Soliris in $2.1B buy­out deal — but will an in­creas­ing­ly vig­i­lant FTC sign off?

UCB is buying out Ra Pharma $RARX, announcing an acquisition deal that rings up at $48 a share, or $2.1 billion net of cash, and puts them toe-to-toe with Alexion on a clinical showdown.

Ra shares closed at $22.70 on Wednesday.

There’s a small pipeline in play at Ra, but UCB is going for the lead drug — a C5 inhibitor called zilucoplan in Phase III for myasthenia gravis (MG) looking to play rival to Alexion’s Soliris. Soliris has the market advantage, though, with a much earlier approval in MG in late 2017 that UCB feels confident in challenging.

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A new play­er is tak­ing the field in a push for a he­mo­phil­ia A gene ther­a­py, and it’s a big one

BioMarin, the execs at Spark (and buyer-to-be Roche) as well as the Sangamo/Pfizer team have a new rival striding onto the hemophilia block. And it’s a big one.

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Stuck with a PhI­II gene ther­a­py fail­ure at 96 weeks, Gen­Sight prefers the up­beat as­sess­ment

Two years after treatment, the best thing that GenSight Biologics $SIGHT can say about their gene therapy for vision-destroying cases of Leber Hereditary Optic Neuropathy is that it’s just a bit better than a placebo — just maybe because one treatment can cover both eyes.

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George Scangos / Credit: Cornell University

ARCH, Soft­Bank-backed Vir Biotech­nol­o­gy un­der­whelms with $143 mil­lion IPO

George Scangos went back to Wall Street, and came back 700 million pennies short.

Scangos’ vaunted startup Vir Biotechnology raised $143 million in an IPO they hoped would earn $150 million. Shares were priced at $20, the low-end of the $20-$22 target.

Launched with backing from ARCH Venture’s Robert Nelsen, Masayoshi Son’s SoftBank Vision Fund, and the Bill & Melinda Gates Foundation, the infectious disease startup was one of a new wave of well-resourced biotechs that emerged with deep enough coffers to pursue a full R&D line rather than slowly build their case by picking off a single lead program.