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.

Un­pack­ing the Aduhelm de­ci­sion, Ver­tex's half full glass, a $525M J&J breakup, 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.

By now you have surely read about the FDA’s controversial approval of Biogen’s Alzheimer’s drug and all its reverberations. But I’d still recommend checking out the meaty recap below to make sure you didn’t miss all the angles that the Endpoints team has covered. If you’d rather look ahead, look no further than our three-day virtual panels next week at BIO, where we will discuss what the new normal means for every part of the industry.

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What does a clear ma­jor­i­ty of the bio­phar­ma in­dus­try think of the FDA ap­proval of ad­u­canum­ab? 'Hor­ri­fy­ing' 'Dan­ger­ous' 'Con­fus­ing' 'Dis­as­ter'

Over the years, we’ve become used to seeing a consensus emerge early in our industry polls at Endpoints News. And when we took the pulse of drug hunters on the heels of a controversial FDA approval for aducanumab this week, it became immediately apparent that the vast majority of our readers — heavily concentrated among biopharma staffers and execs — were incensed by what they had just witnessed.

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Aaron Kesselheim (Scott Eisen/AP Images for AIDS Healthcare Foundation)

Har­vard’s Aaron Kessel­heim re­signs from ex­pert pan­el in wake of ad­u­canum­ab OK, blast­ing FDA for ‘worst drug ap­proval de­ci­sion in re­cent U.S. his­to­ry'

A third member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has resigned in the wake of Biogen’s controversial Aduhelm approval, slamming the agency as he left and further deepening the controversy surrounding the decision.

Harvard University professor Aaron Kesselheim quit in protest Thursday afternoon, calling the Aduhelm OK “probably the worst drug approval decision in recent U.S. history.” Kesselheim follows both Joel Perlmutter, a neurologist from Washington University in St. Louis, and David Knopman, a neurologist from the Mayo Clinic, out the door.

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David Knopman (Mayo Clinic via YouTube)

A sec­ond ad­comm mem­ber aban­dons his post in af­ter­math of con­tro­ver­sial ad­u­canum­ab de­ci­sion

As the fallout from the FDA’s approval of Alzheimer’s med aducanumab grows, a second member of the adcomm overseeing that drug’s review has walked away. But even with two experts now having resigned from that committee in protest, is there enough broad-level outrage to prevent another aducanumab from getting approved?

The FDA on Wednesday lost another member of its Peripheral and Central Nervous System Drugs Advisory Committee as Mayo Clinic neurologist David Knopman hit the exit over the agency’s decision to approve Biogen’s Alzheimer’s drug Aduhelm despite the committee’s near-unanimous vote against it.

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Ver­tex and CRISPR Ther­a­peu­tics un­veil more pos­i­tive gene ther­a­py da­ta, but busul­fan again casts a shad­ow over the field

Less than 12 hours after revealing a flop on its second shot for alpha-1 antitrypsin deficiency, Vertex plowed ahead with another data drop from its partnership with CRISPR Therapeutics. And though the topline proved positive, concerns over conditioning agents continue to linger over the collaboration, as well as the entire gene therapy space.

Presenting data from two trials at the European Hematology Association annual meeting, the pair announced that follow-up data of at least three months for 22 patients with genetic blood disorders indicated a “consistent and sustained” response to the experimental drug CTX001. All 15 patients with transfusion-dependent beta thalassemia did not need further blood transfusions and all seven with severe sickle cell disease were pain free, the biotechs announced.

Reshma Kewalramani, Vertex CEO (BIO via YouTube)

UP­DAT­ED: Ver­tex strikes out on its lat­est big shot at a rare ge­net­ic dis­ease. But they're go­ing to keep on swing­ing

It’s been several months since Vertex culled one of its small molecules for alpha-1 antitrypsin deficiency (AATD), taking a big hit after evidence of liver damage surfaced in a key Phase II trial. Now we learned that the company has whiffed on its second shot, and there’s nothing left in the clinic to treat the rare genetic disease — but that won’t stop it from trying.

Despite avoiding the safety issues that plagued the last candidate, Vertex $VRTX is taking the axe to VX-864 after Phase II results revealed the magnitude of the drug’s response is “unlikely to translate into substantial clinical benefit.” As a result of the news, the company’s stock fell 12.5% after hours.

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FDA au­tho­rizes about 10M J&J vac­cine dos­es, trash­es 60M more from trou­bled Emer­gent plant

The FDA on Friday released about 10 million doses of J&J’s vaccine for use, and disposed of another 60 million doses that were manufactured at the now-shuttered Emergent BioSolutions facility in Baltimore where cross-contamination occurred.

The agency said it’s not yet ready to allow the Emergent plant to be included in the J&J EUA, but that may occur soon. FDA came to the decision to authorize some of the doses after reviewing facility records and quality testing results.

Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

Months af­ter FDA re­jec­tion, Sanofi touts piv­otal win for rare dis­ease drug su­tim­limab as it preps to re­file

One of the pillar drugs of Sanofi’s $11.6 billion pickup of Bioverativ hit a big setback late last year when the FDA sent its application for approval back. Now, as Sanofi gears up to resubmit the drug for review, the drugmaker is touting pivotal data it hopes will help take it over the finish line.

Sanofi’s sutimlimab nailed all three of its primary endpoints in its Phase III CADENZA study for patients with cold agglutinin disease, a rare disorder that can cause severe anemia, without a recent history of blood transfusion, the French drugmaker said Friday. The topline results will be presented at this weekend’s virtual EHA meeting.

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Bris­tol My­er­s' CAR-T Breyanzi busts out a win in ear­li­er-line lym­phoma, po­ten­tial­ly crack­ing open an ex­pand­ed mar­ket

Despite being third to the field in B cell lymphoma, Bristol Myers Squibb has repeatedly argued its CAR-T Breyanzi could have the juice to overtake its older competitors. Going into earlier lines of therapy may be the golden ticket on that front, and now Breyanzi has a late-stage win to back up that effort.

Bristol Myers’s Breyanzi beat out physicians’-choice salvage therapy followed by high-dose chemo and a stem cell transplant — what the drugmaker called a “gold standard treatment” — in second-line patients with relapsed or refractory large B cell lymphoma, according to topline data from the Phase III TRANSFORM study released Thursday.