Edg­ing to­ward a piv­otal read­out, Bio­gen picks a new dis­cov­ery part­ner for oph­thalmic gene ther­a­py

Al­most two years af­ter Bio­gen swooped again in­to the oph­thalmic gene ther­a­py field with an $800 mil­lion buy­out of eye-fo­cused Night­star Ther­a­peu­tics, it’s turn­ing to a young play­er for a new pro­gram.

Ger­many’s Vi­Gen­eron will be de­ploy­ing its next-gen­er­a­tion AAV vec­tors to gen­er­ate can­di­dates for a tar­get — Bio­gen isn’t dis­clos­ing which ex­act­ly — that would treat in­her­it­ed eye dis­ease. The big biotech part­ner has an­oth­er tar­get in mind, for which it has the op­tion to add with­in two years.

Al­though the Alzheimer’s drug ad­u­canum­ab has been shoved to a cen­tral, al­most ex­is­ten­tial po­si­tion at Bio­gen, the com­pa­ny has been plac­ing mea­sured bets on oph­thal­mol­o­gy, which CEO Michel Vounatsos has called an emerg­ing area and part of the “sec­ond wave of growth” to fol­low ad­u­canum­ab in the mid-2020s.

With the new deal, it’s buy­ing in­to a plat­form that boasts of “su­pe­ri­or trans­duc­tion ef­fi­cien­cy” in reti­nal cells and in­trav­it­re­al ad­min­is­tra­tion.

Once it’s come up with the AAV cap­sids, Vi­Gen­eron will play a role in the in vi­vo proof of con­cept, but leave it all to Bio­gen af­ter that point. Bio­gen is pay­ing an undis­closed up­front and foot­ing the R&D bill.

Car­o­line Man Xu

“This col­lab­o­ra­tion ex­em­pli­fies our strat­e­gy to de­vel­op in-house pro­grams for se­lect­ed reti­nal tar­gets, while max­i­miz­ing our pro­pri­etary tech­nol­o­gy plat­forms with ad­di­tion­al col­lab­o­ra­tion pro­grams for oth­er tar­gets in oph­thal­mol­o­gy and fur­ther in­di­ca­tions,” said Car­o­line Man Xu, co-founder and CEO of Vi­Gen­eron.

There’s no word yet on when the can­di­dates might ma­te­ri­al­ize, but they will like­ly come long af­ter Bio­gen reads out the Phase III study of BI­IB111 (tim­repi­gene em­par­vovec), the lead as­set from the Night­star buy­out. De­signed to re­place the gene that’s mu­tat­ed in pa­tients with choroi­deremia, the pro­gram “rep­re­sents our next piv­otal read­out and our next po­ten­tial com­mer­cial prod­uct af­ter ad­u­canum­ab,” Vounatsos said back in Ju­ly.

The da­ta are ex­pect­ed to come in the first half of 2021.

Aside from that and a sec­ond Night­star ex­per­i­men­tal treat­ment for re­tini­tis pig­men­tosa caused by mu­ta­tions in the RP­GR gene, Bio­gen has al­so picked up a pre­clin­i­cal gene ther­a­py from Mass­a­chu­setts Eye and Ear for in­her­it­ed reti­nal de­gen­er­a­tion due to mu­ta­tions in the PRPF31 gene.

It all marks a sec­ond try at de­vel­op­ing one-shot cures for rare eye dis­eases, af­ter pulling out from a deal with Ap­plied Ge­net­ic Tech­nolo­gies fol­low­ing a failed tri­al.

Vac­cine doc­u­ments, young lead­ers and mar­ket tur­moil: End­points' 10 biggest sto­ries of 2022

It’s been a volatile year in the world of biopharma. Market declines reset M&A valuations, and may be beginning to tempt bigger buyers back into dealmaking. Russia’s war in Ukraine disrupted drug sales and clinical trials. A new generation of young biotech leaders emerged in the Endpoints 20(+1) Under 40. And as capital runs dry in a tough environment for raising new funds, companies big and small are taking a look at their headcounts and operations for ways to make it through lean times.

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Tom Riga, Spectrum Pharmaceuticals CEO

Spec­trum im­plodes af­ter a harsh pub­lic slap­down and now a CRL from Richard Paz­dur

The FDA has gone out of its way several times to flatten any expectations for Spectrum’s lung cancer drug poziotinib, including slamming the regulatory door in the biotech’s face four years ago when the their executive crew came calling for a breakthrough drug designation and encouragement from the oncology wing of the FDA.

That stinging early rebuke pointed straight down the path to a corrosive in-house agency review of Spectrum’s attempt to land an accelerated approval for the oral EGFR TKI and a public whipping that included a classic takedown by none other than Richard Pazdur, who slammed the company for “poor drug development” that led to confusion over the dose needed for a slice of NSCLC patients harboring HER2 exon 20 insertion mutations.

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Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; 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.

As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

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Albert Bourla, Pfizer CEO (John Thys/POOL/AFP via Getty Images)

Pfiz­er CEO un­der fire from UK watch­dog over vac­cine com­ments — re­port

Pfizer CEO Albert Bourla told the BBC last December that he had “no doubt in my mind that the benefits, completely, are in favor” of vaccinating 5- to 11-year-olds for Covid-19. Almost a year later, those comments have reportedly landed him in trouble with a UK pharma watchdog.

Children’s advocacy group UsForThem filed a complaint with the UK’s Prescription Medicines Code of Practice Authority (PMCPA) last year accusing Bourla of making “disgracefully misleading” statements during the BBC interview, including one that “Covid in schools is thriving.” At the time, UK regulators had not yet cleared the vaccine for the 5 to 11 age group, though the vaccine did have a positive opinion from the EMA’s human medicines committee.

FDA tells Catal­ent to fix is­sues at two man­u­fac­tur­ing sites on its own

The CDMO Catalent will have to fix issues at two manufacturing plants in the US and Europe that were subject to inspections by the FDA this summer, giving the company room to correct the issues without facing further regulatory action.

The FDA gave Catalent a “voluntary action indicated” response to two inspections at the contract manufacturer’s site in Bloomington, IN, and Brussels, Belgium. Fixing the issues on its own is a preferable outcome to facing an “official action indicated” response, meaning that an official warning would be sent out or a sit-down with the FDA would be required.

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Merck targets vaccine-hesitant parents in its latest 'Why Vaccines' campaign. (Image: Shutterstock)

Mer­ck­'s lat­est 'Why Vac­ci­nes' cam­paign seeks to bet­ter in­form vac­cine-hes­i­tant moms

From Hollywood couple endorsements to targeted equity efforts, Merck has been pushing the value of vaccinations, especially since the Covid-19 pandemic disruption. Now the pharma is turning to a new target — vaccine-hesitant parents, and moms in particular.

Merck’s “Why Vaccines” latest social media and digital campaign spotlights real-life new moms who have questions about vaccinating their children.

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In­dus­try groups, CVS pick apart FDA's pro­posed path­way for gener­ics to carve out OTC in­di­ca­tions

Pharma industry groups like the Association for Accessible Medicines (AAM) and PhRMA are raising pointed questions about an FDA plan to create a new pathway for marketing prescription drugs with an additional condition for nonprescription use (ACNU), which would require more safeguards than the current OTC pathway but essentially carve out new OTC uses for some generic drugs.

Chief among the concerns were: Insurance companies dropping coverage for the Rx version, new ACNU patents to block competition, and industry essentially governing the pathway.

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Robert Califf, FDA commissioner (Jose Luis Magana/AP Images)

Fourth ac­cel­er­at­ed ap­proval in Duchenne? Sarep­ta gets pri­or­i­ty re­view for gene ther­a­py amid FDA scruti­ny

Sarepta is once again on the accelerated approval path for a Duchenne drug, picking up a priority review Monday morning.

The FDA granted the accelerated review to SRP-9001, Sarepta announced Monday, which would become the biotech’s fourth Duchenne drug if approved. Much like SRP-9001 will do, each of the previous three therapies went through the accelerated approval pathway. But unlike the others, SRP-9001 is a gene therapy.

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Mene Pangalos, AstraZeneca EVP of BioPharmaceuticals R&D (AstraZeneca via YouTube)

In new deal, As­traZeneca places small bet on 'crit­i­cal but chal­leng­ing' tar­get

AstraZeneca has its eyes on NRF2 as a target for inflammatory and respiratory diseases, and it’s handing out $2 million in cash to pluck a preclinical candidate from a drug discovery shop.

It’s so early that C4X Discovery doesn’t have a name for the oral NRF2 activator program just yet. Including the upfront, the biotech is eligible for a total of $16 million in preclinical payments, after which AstraZeneca will take over for clinical development and commercialization. Those future milestones add up to $385.8 million.

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