Joe Nolan, Jaguar

Sean Nolan gath­ers AveX­is alum­ni to launch an­oth­er biotech. Is it an­oth­er race-to-IPO?

The old AveX­is crew is launch­ing an­oth­er com­pa­ny. And they may once again look to cash in quick.

Armed with patents and a steady stream of cash from Deer­field Man­age­ment, Sean Nolan and five ex-AveX­is ex­ec­u­tives have launched Jaguar Gene Ther­a­py to de­vel­op AAV9 gene ther­a­pies for a small con­stel­la­tion of dis­parate dis­or­ders, from autism to di­a­betes. Jaguar re­mains over a year from the clin­ic, but CEO Joe Nolan hint­ed they could look to fol­low a pace set by Ta­sy­ha, the biotech Sean Nolan and oth­er AveX­is alum­ni launched last year and took from Se­ries A to IPO in less than 6 months.

Sean Nolan

“It’s hard to say, ob­vi­ous­ly we’re re­al­ly fo­cused on what we’re do­ing, but I do think we can quick­ly move to oth­er fund­ing sources,” Joe Nolan (no re­la­tion to Sean) told End­points News. “I don’t know if we’ll get to IPO that fast, but, you know, we’ll see.”

The idea for Jaguar came from Deer­field, who had as­sem­bled a col­lec­tion of in­tel­lec­tu­al prop­er­ty on gene ther­a­py and went look­ing for an ex­ec­u­tive team around the time many of the for­mer AveX­is ex­ec­u­tives were leav­ing No­var­tis. Deer­field has since pumped around $40 mil­lion in­to the com­pa­ny as Jaguar has ex­pand­ed to a team of about 30 in Lake For­est, Illi­nois and North Car­oli­na.

Their gene ther­a­py for a ge­net­ic cause of autism came out of one of the col­lab­o­ra­tions Deer­field has been sign­ing with uni­ver­si­ties across the coun­try over the last few years. And last year Deer­field li­censed a gene ther­a­py for the rare neu­ro-meta­bol­ic dis­ease Bardet-Biedl Syn­drome from the Uni­ver­si­ty of Col­lege Lon­don. That ther­a­py will be de­vel­oped by Ax­ovia Ther­a­peu­tics, a ma­jor­i­ty-owned sub­sidiary of Jaguar.

So far, though, the com­pa­ny is re­main­ing tight-lipped on specifics. Nolan wouldn’t say which of the sev­er­al po­ten­tial ge­net­ic tar­gets they would go af­ter in autism or even which uni­ver­si­ty it came from. Nor would he dis­close the tar­get they’ll go af­ter for type 1 di­a­betes — a dis­ease aca­d­e­mics have the­o­rized tar­get­ing with gene ther­a­py in a va­ri­ety of ways — or where they li­censed that ther­a­py from.

The com­pa­ny’s pitch rests large­ly on AveX­is’s rep­u­ta­tion in AAV gene ther­a­py. Nolan said they bring ex­per­tise from de­vel­op­ment to man­u­fac­tur­ing and com­mer­cial­iza­tion.

“We have a lot of ex­pe­ri­ence,” Nolan said. “We be­lieve it’s a proven and re­li­able way to min­i­mize de­vel­op­ment risk, we have a suc­cess­ful track record there and we think we know the hur­dles ahead be­cause we’ve been through it be­fore.”

The AAV field has tak­en a hit in the last year, af­ter the death of three pa­tients in one high-dose Au­dentes tri­al and pos­si­ble safe­ty sig­nal in oth­ers. Nolan said they viewed those is­sues as be­ing spe­cif­ic to those spe­cif­ic con­structs as op­posed to a broad­er is­sue with AAV gene ther­a­py, though he added that the com­pa­ny wouldn’t be push­ing the dose as high as Au­dentes had.

“There’s an art to not push­ing the dose, par­tic­u­lar­ly in IV,” he said.

Suku­mar Na­gen­dran

Nolan, an Ab­bot and Take­da vet­er­an, was hired in 2018 as a gen­er­al man­ag­er at AveX­is, where he helped de­sign the com­mer­cial launch plan for Zol­gens­ma. He says he keeps a pic­ture of the first kids they treat­ed on his night­stand, next to pic­tures of his own. Suku­mar Na­gen­dran, who was CMO at AveX­is, has joined as an R&D chief. Sean Nolan is chair­man of the board.

The first ther­a­py they plan to put in the clin­ic is a gene ther­a­py for Bardet-Biedl Syn­drome, with an IND planned for the sec­ond half of 2022.

Should we ex­pect Sean Nolan to launch more biotechs in the mean­time?

“I don’t think so,” Joe Nolan said, be­fore adding: “Sean’s ob­vi­ous­ly pop­u­lar, I can’t speak to his fu­ture, but I can say he is ex­cit­ed about this one.”

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,200+ biopharma pros reading Endpoints daily — and it's free.

Ap­peals court puts the fi­nal nail in the cof­fin for Tec­fidera patent, adding to Bio­gen's bur­geon­ing set­backs

In another setback for Biogen, the big biotech lost its appeal to revive a patent for the once-blockbuster drug Tecfidera, marking a likely conclusion to the case.

The US Court of Appeals for the Federal Circuit issued the ruling Tuesday morning, saying Biogen failed to satisfy the “written description” requirement for patent law. As a result, Mylan-turned-Viatris will be able to sell its multiple sclerosis generic without fear of infringement and Biogen will have to find a new revenue driver elsewhere.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,200+ biopharma pros reading Endpoints daily — and it's free.

Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,200+ biopharma pros reading Endpoints daily — and it's free.

In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

As lead drug runs in­to a wall, De­ci­phera slims down its pipeline, puts 140 jobs on the chop­ping block

Barely a month after disappointing data shattered hopes for a major label expansion for the GI tumor drug Qinlock, Deciphera is making a major pivot — scrapping development plans for that drug and discarding another while it hunkers down and focuses on two remaining drugs in the pipeline.

As a result, 140 of its staffers will be laid off.

The restructuring, which claims the equivalent of 35% of its total workforce, will take place across all departments including commercial, R&D as well as general and administrative support functions, Deciphera said, as it looks to streamline Qinlock-related commercial operations in the US while concentrating only on a “select number of key European markets.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,200+ biopharma pros reading Endpoints daily — and it's free.

How to use reg­istry da­ta to sup­port FDA de­ci­sion mak­ing: Agency ex­plains in new guid­ance

Drugmakers looking to design a new registry or use an existing one to support a regulatory decision on a drug’s effectiveness or safety will need to consult with a new draft guidance released Monday by the FDA.

The agency’s reliance on registry data for regulatory decisions dates back more than two decades, at least, as in 1998 Bayer won approval for its anticoagulant Refludan (withdrawn from the market in 2013 for commercial reasons) based in part on a historical control group pulled from a registry.

Tillman Gerngross (Adagio)

Till­man Gern­gross on Omi­cron: 'It is a grim sit­u­a­tion...we’re go­ing to see a sig­nif­i­cant drop in vac­cine ef­fi­ca­cy'

Tillman Gerngross, the rarely shy Dartmouth professor, biotech entrepreneur and antibody expert, has been warning for over a year that the virus behind Covid-19 would likely continue to mutate, potentially in ways that avoid immunity from infection and the best defenses scientists developed. He spun out a company, Adagio, to build a universal antibody, one that could snuff out any potential mutation.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.