Hunt­ing a cure, Spark re­ports steady progress on he­mo­phil­ia B gene ther­a­py as Pfiz­er preps for piv­otal hand­off

Af­ter some fine tun­ing on the man­u­fac­tur­ing side, Spark Ther­a­peu­tics $ONCE says it’s racked up a new round of promis­ing re­sults for its Phase I/II study of a ground­break­ing new gene ther­a­py that aims to cure he­mo­phil­ia B. And the biotech is prep­ping a hand­off of SPK-9001 to its Big Phar­ma al­lies at Pfiz­er $PFE this sum­mer, who are plan­ning to start a Phase III piv­otal study.

Kather­ine High

Re­searchers are now re­port­ing on 15 pa­tients in the ear­ly-stage study get­ting a re­place­ment fac­tor IX gene to cor­rect the mu­ta­tion that re­quires con­stant care and fac­tor re­place­ment ther­a­py to pre­vent bleeds. The an­nu­al­ized bleed rate for all of them was cut by 97% and re­searchers were care­ful to high­light three pa­tients who are do­ing fine — with fac­tor IX lev­els rang­ing from 38.1% to 54.5% be­yond the 12-week mark— af­ter be­ing treat­ed with an en­hanced ther­a­py pro­duced by a new man­u­fac­tur­ing ap­proach.

The an­nu­al in­fu­sion rate was slashed to 0.9 in­fu­sions, com­pared to 57.2 in­fu­sions be­fore the study. The da­ta were be­ing pre­sent­ed at the World Fed­er­a­tion of He­mo­phil­ia World Con­gress in Glas­gow to­day.

For the full group in the Spark study, the range on fac­tor IX was 14.3% to 76.8%. Six of the 15 had to get in­fu­sions; two for spon­ta­neous bleeds, two pri­or to surgery, and one each for a mi­nor trau­ma and at the end of the study pe­ri­od.

Elim­i­nat­ing these in­fu­sions safe­ly and re­li­ably will be key for Spark and Pfiz­er as they look to field the first he­mo­phil­ia gene ther­a­py, rac­ing against Bio­Marin $BM­RN and uniQure. Sang­amo $SG­MO, mean­while, is us­ing its gene edit­ing tech for he­mo­phil­ia B. 

Spark gained an his­toric FDA OK for Lux­tur­na, the first gene ther­a­py ap­proved in the US, which came out with a list price of $850,000. But an­a­lysts say that a he­mo­phil­ia gene ther­a­py could be the first to be mar­ket­ed for as much as $1.5 mil­lion, con­sid­er­ing the steep cost of in­fu­sions pa­tients un­der­go.

“We are pleased to see all 15 par­tic­i­pants, no­tably in­clud­ing the first four par­tic­i­pants who have been fol­lowed for more than two years, con­tin­ue to show that a sin­gle ad­min­is­tra­tion of SPK-9001 has re­sult­ed in dra­mat­ic re­duc­tions in bleed­ing and fac­tor IX in­fu­sions, with no se­ri­ous ad­verse events,” said Kather­ine High, the pres­i­dent and head of re­search & de­vel­op­ment at Spark Ther­a­peu­tics.

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.