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.

In a sec­ond big set­back for Covid-19 an­ti­body treat­ment hopes, Re­gen­eron halts en­roll­ment for more se­vere pa­tients

Regeneron has just delivered more bad news for the hope that neutralizing antibodies could be used to treat patients with more severe forms of Covid-19.

The New York biotech said today that an independent monitoring committee recommended halting enrollment of patients who need high-flow oxygen or mechanical ventilation in one of the trials on their antibody cocktail, after finding “a potential safety signal” and “an unfavorable risk/benefit profile.” The news comes a week after the NIH scrapped a trial of Eli Lilly’s Covid-19 antibody after finding it was having little effect on an initial cohort of hospitalized patients.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,900+ biopharma pros reading Endpoints daily — and it's free.

Daphne Koller, Getty

Bris­tol My­er­s' Richard Har­g­reaves pays $70M to launch a neu­rode­gen­er­a­tion al­liance with a star play­er in the ma­chine learn­ing world

Bristol Myers Squibb is turning to one of the star upstarts in the machine learning world to go back to the drawing board and come up with the disease models needed to find drugs that can work against two of the toughest targets in the neuro world.

Daphne Koller’s well-funded insitro is getting $70 million in cash and near-term milestones to use their machine learning platform to create induced pluripotent stem cell-derived disease models for ALS and frontotemporal dementia.

CEO Kenji Yasukawa (Astellas)

In ear­ly blow to Ken­ji Ya­sukawa's R&D re­vamp, Astel­las drops out of the TIG­IT race, cit­ing PhI fail­ure

Just after AstraZeneca jumped into the TIGIT race, Astellas quietly disclosed that it was leaving, dropping out of a hunt for an immunotherapy approach that has shown tantalizing promise but remains largely unproven.

Astellas revealed in their second quarter earnings today that they’ve ended development of the anti-TIGIT antibody they acquired in their up to $400 million buyout of Potenza in 2018. The Japanese pharma had been testing it in combination with Keytruda in a 300-person Phase I study on patients with advanced solid tumors. A smaller study testing the antibody alone was completed, 2 years ahead of schedule, in July.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,900+ biopharma pros reading Endpoints daily — and it's free.

Bel­licum slash­es 79% of staffers af­ter ear­ly da­ta quash hope around next-gen CAR-T

Bellicum Pharmaceuticals tried to make it work.

Over the past months the Houston-based CAR-T player sold its manufacturing facility to MD Anderson — transferring 35 employees in the process — in favor of an outsourced arrangement and completed a reverse stock split, while dribbling out new data and getting an IND cleared.

But new interim data from a Phase I/II trial provided the last straw. Bellicum disclosed late Thursday that it will be laying off 79% of its staff, shaving the workforce from 68 to just 14 by the end of 2020.

George Golumbeski (L) and Faheem Hasnain

George Golumbes­ki and Fa­heem Has­nain team up with Ver­tex Ven­tures HC in man­ag­ing $320M of biotech cash

Two longtime biotech veterans are joining a multibillion dollar VC firm in order to help steer its latest fund.

George Golumbeski and Faheem Hasnain have signed on to Vertex Ventures HC as executive advisors, the company announced Thursday, and will assist with their depth of experience in managing $320 million of capital. Both have had previous working relationships with managing partners Carolyn Ng and Lori Hu, which evolved “organically” to get to this point, Ng said.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,900+ biopharma pros reading Endpoints daily — and it's free.

Patrick Soon-Shiong at the JP Morgan Healthcare Conference, Jan. 13, 2020 (David Paul Morris/Bloomberg via Getty Images)

Af­ter falling be­hind the lead­ers, dissed by some ex­perts, biotech show­man Patrick Soon-Sh­iong fi­nal­ly gets his Covid-19 vac­cine ready for a tri­al. But can it live up to the hype?

In January, when dozens of scientists rushed to start making a vaccine for the then-novel coronavirus, they were joined by an unlikely compatriot: Patrick Soon-Shiong, the billionaire doctor most famous for making big, controversial promises on cancer research.

Soon-Shiong had spent the last 4 years on his “Cancer Moonshot,” but part of his project meant buying a small Seattle biotech that specialized in making common-cold vectors, called adenoviruses, to train the immune system. The billionaire had been using those vectors for oncology, but the company had also developed vaccine candidates for H1N1, Lassa fever and other viruses. When the outbreak began, he pivoted.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,900+ biopharma pros reading Endpoints daily — and it's free.

Eli Lilly CEO David Ricks (Evan Vucci/AP Images)

A p-val­ue of 0.38? NE­JM re­sults raise new ques­tions for Eli Lil­ly's vaunt­ed Covid an­ti­body

Generally, a p-value of 0.38 means your drug failed and by a fair margin. Depending on the company, the compound and the trial, it might mean the end of the program. It could trigger layoffs.

For Eli Lilly, though, it was part of the key endpoint on a trial that landed them a $1.2 billion deal with the US government to supply up to nearly 1 million Covid-19 antibodies.

So what does one make of that? Was the endpoint not so important, as Lilly maintains? Or did the US government promise a princely sum for a pedestrian drug?

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,900+ biopharma pros reading Endpoints daily — and it's free.

No­var­tis buys a new gene ther­a­py for vi­sion loss, and this is one pre­clin­i­cal ven­ture that did­n't come cheap

Cyrus Mozayeni got excited when he began to explore the academic work of Ehud Isacoff and John G. Flannery at UC Berkeley.

Together, they were engaged in finding a gene therapy approach to pan-genotypic vision restoration in patients with photoreceptor-based blindness, potentially restoring the vision of a broad group of patients. And they did it by using a vector to deliver the genetic sequence for light sensing proteins.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,900+ biopharma pros reading Endpoints daily — and it's free.

CMO Merdad Parsey (Gilead)

Gilead hits the brakes on a tri­fec­ta of mid- and late-stage stud­ies for their trou­bled fil­go­tinib pro­gram. It's up to the FDA now

Gilead $GILD execs haven’t decided exactly what to do with filgotinib in the wake of the slapdown at the FDA on their rheumatoid arthritis application, but they’re taking a time out for a slate of studies until they can gain some clarity from the agency. And without encouraging guidance, this drug could clearly be axed from the pipeline.

In their Q3 report out Wednesday afternoon, the company says researchers have “paused” a Phase III study for psoriatic arthritis along with a pair of Phase II trials for ankylosing spondylitis and uveitis. Late-stage studies for ulcerative colitis and Crohn’s are continuing, but you can see for yourself how big a hole this leaves in the inflammatory disease pipeline, with obvious implications if the company abandons filgo altogether.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,900+ biopharma pros reading Endpoints daily — and it's free.