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.

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His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

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Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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Seagen interim CEO Roger Dansey and Daiichi Sankyo CEO Sunao Manabe

Paving the way for Mer­ck­'s buy­out, Seagen los­es ar­bi­tra­tion dis­pute with Dai­ichi over ADC tech

As Seagen awaits a final buyout offer from Merck that could be in the territory of $40 billion, Seagen revealed Friday afternoon that it lost an arbitration dispute with Daiichi Sankyo relating to the companies’ 2008 collaboration around the use of antibody-drug conjugate (ADC) technology.

But that loss likely won’t matter much when it comes to Merck’s deal.

After breaking off its pact with Daiichi in mid-2015, the two companies battled over “linker” tech — a chemical bridge between an ADC’s antibody component and the cytotoxic payload — that Seagen claims Daiichi would improve upon and implement in its current generation of ADCs.

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Benjamin Oakes, Scribe Therapeutics CEO

CEO of Doud­na spin­out: With­in five years, genome ed­i­tors will have a 're­al­ly big im­pact' on pa­tients' lives

“CRISPR-by-design” is the idea behind Scribe Therapeutics, a company spun out from Jennifer Doudna’s Nobel-winning lab that’s competing in a closely-tracked field of genome editor companies just starting to make their way to the clinic.

After nabbing $100 million last March for its Series B funding round, Scribe is taking a different tack from some of its competitors, crafting a new enzyme isolated from bacteria called CasX, which has now been tweaked extensively and may be targeted to a range of genome-related diseases, offering a plethora of therapeutic options.

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House pass­es his­toric drug pric­ing re­forms, lin­ing up decades-in-the-mak­ing win for Biden and De­moc­rats

The US House of Representatives today voted along party lines (all Dems voted for it), 220-207 to pass new, wide-ranging legislation that will allow Medicare drug price negotiations for the first time ever, and cap seniors’ drug expenses to $2,000 per year and seniors’ insulin costs at $35 per month.

Setting up a major victory for President Joe Biden, representatives returned from their summer recess to pass the Inflation Reduction Act, even as many noted the bill would only modestly reduce inflation.

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Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

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J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.