Voy­ager Ther­a­peu­tics adds proof its gene ther­a­py for Parkin­son's can work, shares soar

Voy­ager Ther­a­peu­tics $VY­GR says it’s turned an im­por­tant cor­ner in its de­vel­op­ment of a gene ther­a­py that can play a ma­jor role in res­cu­ing Parkin­son’s pa­tients from the in­evitable de­cline as­so­ci­at­ed with their re­sponse to lev­odopa.

The Cam­bridge, MA-based biotech has fleshed out its ear­ly-stage proof-of-con­cept da­ta on VY-AADC01, of­fer­ing new da­ta that demon­strate a height­ened ef­fi­ca­cy for two high­er dos­es of the gene ther­a­py. Ex­ec­u­tives at Voy­ager tell me that they’re heart­ened to see a dose-de­pen­dent re­sponse in a key bio­mark­er on their ther­a­py’s im­pact, with im­prove­ments in hourly “on” times, a drop in “off” times and bet­ter qual­i­ty of liv­ing scores from the 15 pa­tients di­vid­ed in­to three ther­a­peu­tic co­horts.

A low dose of the gene ther­a­py paled in com­par­i­son to the two oth­er co­horts in the study, which did not in­clude a place­bo group.

Voy­ager joined a crowd of biotechs in the win­ners cir­cle on Wall Street this morn­ing, with its shares – up about 10% yes­ter­day – adding an 36% spike Wednes­day.

Voy­ager CEO Steven Paul

“Co­hort 2 con­tin­ues to look great,” Voy­ager CEO Steve Paul tells me. “We need to wait to get Co­hort 3 to 12 months to choose the dose for the piv­otal tri­al.”

But it’s com­ing. And with it, Voy­ager is map­ping plans to ex­e­cute a tri­al that can be used to seek an ac­cel­er­at­ed ap­proval.

There are all sorts of caveats that ap­ply to this da­ta, aside from the lack of a place­bo arm. The num­bers of pa­tients in­volved in this lat­est up­date, which fol­lows the first round of pos­i­tive re­sults from the ear­ly tri­al, re­mains small. A piv­otal will be much more de­mand­ing. And Voy­ager re­searchers — who ear­li­er record­ed a blood clot case in the study — still has to prove that the process can be done com­plete­ly safe­ly.

But now there’s more sol­id da­ta to un­der­score that Voy­ager’s ap­proach has promise, a rare event in Parkin­son’s, one of the tough­est dis­eases in biotech. And they say that a move to in­sert the gene ther­a­py through the back of the head ap­pears safe and eas­i­er to com­plete.

The gene ther­a­py is de­signed to com­plete a sim­ple task. Parkin­son’s pa­tients typ­i­cal­ly re­spond well to lev­odopa to pro­vide the dopamine pa­tients need fol­low­ing the death of neu­rons in the brain. But their re­sponse de­clines, re­quir­ing ever high­er dos­es of lev­odopa with ever di­min­ish­ing re­turns. Voy­ager’s gene ther­a­py in­tro­duces an en­zyme that con­verts lev­odopa to dopamine, and this study un­der­scores that pa­tients were able to get a bet­ter ef­fect with low­er dos­ing — a ma­jor ac­com­plish­ment.

“What we’re do­ing is putting the gene for that en­zyme in neu­rons that are still alive and healthy, ar­ti­fi­cial­ly al­low­ing the brain re­gion to con­tin­ue mak­ing dopamine,” says Paul, a long­time Eli Lil­ly vet be­fore he jumped in­to biotech.

Those dead neu­rons, adds Paul, aren’t com­ing back to life. Voy­ager looks at this as a restora­tive strat­e­gy, which in pri­mates has proved ef­fec­tive 15 years out.

The plan now is to launch a po­ten­tial­ly piv­otal tri­al with 40-42 pa­tients in the first half of next year, point­ing to a pos­si­ble BLA if reg­u­la­tors sign off.

Paul says Voy­ager is right on track dur­ing a key tran­si­tion point for his com­pa­ny, one of a wave of gene ther­a­py biotechs that gained the spot­light in re­cent years. As Di­men­sion’s prob­lems with he­mo­phil­ia B proved, along with its re­cent sale to Re­gen­rx, not all these com­pa­nies will make it through. But Voy­ager is still very much in the game.

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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South Ko­rea jails 3 Sam­sung ex­ecs for de­stroy­ing ev­i­dence in Bi­o­Log­ics probe

Three Samsung executives in Korea are going to jail.

The convictions came in what prosecutors had billed as “biggest crime of evidence destruction in the history of South Korea”: a case of alleged corporate intrigue that was thrown open when investigators found what was hidden beneath the floor of a Samsung BioLogics plant. Eight employees in total were found guilty of evidence tampering and the three executives were each sentenced to up to two years in prison.

Nick Plugis, Avak Kahvejian, Cristina Rondinone, Milind Kamkolkar and Chad Nusbaum. (Cellarity)

Cel­lar­i­ty, Flag­ship's $50M bet on net­work bi­ol­o­gy, mar­ries ma­chine learn­ing and sin­gle-cell tech for drug dis­cov­ery

Cellarity started with a simple — but far from easy — idea that Avak Kahvejian and his team was floating around at Flagship Pioneering: to digitally encode a cell.

As he and his senior associate Nick Plugis dug deeper into the concept, they found that most of the models others have developed take a top-down approach, where they assemble the molecules inside cells and the connections between them from scratch. What if they opt for a top-down approach, aided by single-cell transcriptomics and machine learning, to gauge the behavior of the entire cellular network?

Roger Perlmutter, Merck

#ASH19: Here’s why Mer­ck is pay­ing $2.7B to­day to grab Ar­Qule and its next-gen BTK drug, lin­ing up Eli Lil­ly ri­val­ry

Just a few months after making a splash at the European Hematology Association scientific confab with an early snapshot of positive data for their BTK inhibitor ARQ 531, ArQule has won a $2.7 billion buyout deal from Merck.

Merck is scooping up a next-gen BTK drug — which is making a splash at ASH today — from ArQule in an M&A pact set at $20 a share $ARQL. That’s more than twice Friday’s $9.66 close. And Merck R&D chief Roger Perlmutter heralded a deal that nets “multiple clinical-stage oral kinase inhibitors.”

This is the second biotech buyout pact today, marking a brisk tempo of M&A deals in the lead-up to the big JP Morgan gathering in mid-January. It’s no surprise the acquisitions are both for cancer drugs, where Sanofi will try to make its mark while Merck beefs up a stellar oncology franchise. And bolt-ons are all the rage at the major pharma players, which you could also see in Novartis’ recent $9.7 billion MedCo buyout.

ArQule — which comes out on top after their original lead drug foundered in Phase III — highlighted early data on ‘531 at EHA from a group of 6 chronic lymphocytic leukemia patients who got the 65 mg dose. Four of them experienced a partial response — a big advance for a company that failed with earlier attempts.

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Sanofi’s big week in­cludes a promis­ing PhI­II for an or­phan dis­ease drug, with plans for a pitch to the FDA

The biopharma R&D food chain is paying off with a plan at Sanofi to pitch regulators on a new drug for an orphan disease called cold agglutinin disease.

The pharma giant ushered out a statement Tuesday morning — after it spelled out plans to radically restructure the company, abandoning cardio and diabetes research altogether — saying that their C1s inhibitor sutimlimab had cleared the pivotal study.

In­scrip­ta push­es fund­ing to $260 mil­lion as they launch genome edit­ing plat­form

Inscripta presentations can begin with the advent of agriculture. Or even further back: The emergence of man.

“We’ve come a long way, starting with natural selection,” Inscripta microbial director Nandini Krishnamurthy told a session this year at SynBioBeta, the new annual conference for the synthetic biology field.

Behind her was a slide that’s recurred in company presentations, showing from left to right across the bottom the classic evolution-of-man chart (the ‘humorous’ kind that ends on an overweight soda-drinker), a picture showing the development of corn from thin strand to bulbous Iowan, and then a squiggly protein close-up of “directed evolution.” Below that runs an arrow and a ticker of how long each takes, from 10^9 years to 1.

Left top to right: Mark Timney, Alex Denner, Vas Narasimhan. (The Medicines Company, Getty, AP/Endpoints News)

In a play-by-play of the $9.7B Med­Co buy­out, No­var­tis ad­mits it over­paid while of­fer­ing a huge wind­fall to ex­ecs

A month into his tenure at The Medicines Company, new CEO Mark Timney reached out to then-Novartis pharma chief Paul Hudson: Any interest in a partnership?

No, Hudson told him. Not now, at least.

Ten months later, Hudson had left to run Sanofi and Novartis CEO Vas Narasimhan was paying $9.7 billion for the one-drug biotech – the largest in the string of acquisitions Narasimhan has signed since his 2017 appointment.

The deal was the product of an activist investor and his controversial partner working through nearly a year of cat-and-mouse negotiations to secure a deal with Big Pharma’s most expansionist executive. It represented a huge bet in a cardiovascular field that already saw two major busts in recent years and brought massive returns for two of the industry’s most eye-raising names.

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Re­pub­li­cans un­veil a drug price bill to ri­val the De­moc­rats — promis­ing low­er prices and more cures

Nancy Pelosi unveiled the Democrats’  drug pricing bill back in September and brought the fight straight to the industry with a proposal to empower the US government to negotiate prices for select drugs. Republicans, who decried the bill reeks of heavy-handed government intervention which will stifle innovation, now have a counterproposal they claim will result in cheaper drugs and incentivize R&D — further clouding the prospects of a bipartisan compromise that could land on Donald Trump’s desk.