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.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.

We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Mer­ck Serono’s Se­nior Vice Pres­i­dent and Glob­al Head of On­col­o­gy
EL­LIOTT LEVY — Am­gen’s Se­nior Vice Pres­i­dent of Glob­al De­vel­op­ment
CHRIS BOSHOFF — Pfiz­er On­col­o­gy’s Chief De­vel­op­ment Of­fi­cer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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Ver­sant-backed Chi­nook gets a $65M launch round for its dis­cov­ery quest in a resur­gent kid­ney field

Versant is once again stepping off the beaten track in biotech to see if they can blaze a trail of their own in a field that has looked too thorny to many investors for years.

The venture group and their partners at Apple Tree are bringing their latest creation out of stealth mode today. Born in Versant’s Inception Sciences’ Chinook Therapeutics is betting that its preclinical take on kidney disease can get an early lead among the companies starting up in the field.

Sir An­drew Dil­lon, NICE's first — and on­ly — chief ex­ec­u­tive to step down next year

Using a laptop borrowed from his former employer, South London’s St George’s Hospital, Sir Andrew Dillon set about establishing NICE — launched by the then health secretary Frank Dobson — in 1999.  On Thursday, the UK cost-effectiveness watchdog said its first and only chief executive — Dillon — is stepping down in March 2020.

Back in the day, decisions about which drugs and interventions were funded by the National Health Service (NHS) were made at the local level, but this ‘postcode prescribing’ system was fraught with skewed healthcare deployment making the structure unsustainable. A national system was deemed necessary — and NICE was formed to bridge that gap.

Eight weeks be­tween each HIV treat­ment? GSK notch­es PhI­II win as it chas­es OK for long-act­ing reg­i­men

GSK has cleared another test in its grand plan to topple Gilead’s HIV dominance by offering alternative treatments that consist of fewer drugs and last longer. A year after scoring positive Phase III data on a four-week course of cabotegravir and rilpivirine, its ViiV subsidiary now says that an eight-week regimen seem to work just as well.

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Finch grabs a $53M round de­signed to take their ‘break­through’ mi­cro­bio­me treat­ment through a po­ten­tial­ly piv­otal tri­al

With a breakthrough designation in one hand and a fresh $53 million in venture backing in the other, Somerville, MA-based Finch Therapeutics is taking a shot at a one-trial pathway to a possible FDA OK for their new treatment for preventing recurrent C. difficile infections.

The funding brings their total raise for the microbiome company to $130 million, CEO Mark Smith tells me — enough money to pave a runway past the FDA approval they’ve sketched into the most optimistic version for their near-term future.