Can a pair of top AveX­is alum­ni steer a new gene ther­a­py up­start to R&D glo­ry? 3 VCs bet $60M on it

Rick Mo­di

VCs love few things more than a proven ex­ec­u­tive team when it comes to launch­ing a new com­pa­ny. And now a group of A-lis­ters has turned to a pair of top ex­ecs out of AveX­is to steer the lat­est gene ther­a­py play­er in­to the clin­ic.

The biotech is Waltham, MA-based Affinia and the two ex­ecs are Sean Nolan and Rick Mo­di — the for­mer CEO and CBO re­spec­tive­ly of AveX­is, the gene ther­a­py pi­o­neer that fetched $8.7 bil­lion in a sale to No­var­tis. Nolan has now tak­en the chair­man’s role at Affinia while Mo­di moves up to the CEO post at the com­pa­ny.

Sean Nolan

Both were key play­ers on a team that steered Zol­gens­ma to the mar­ket as the most ex­pen­sive once-off drug now avail­able. They worked close­ly with CSO Bri­an Kas­par, who was blamed by No­var­tis for in­clud­ing ma­nip­u­lat­ed da­ta in their mar­ket­ing ap­pli­ca­tion, trig­ger­ing a scan­dal that con­sumed No­var­tis for weeks. Kas­par ve­he­ment­ly de­nied the ac­cu­sa­tion, though, and the scan­dal qui­et­ly dis­si­pat­ed with the FDA tak­ing none of the ac­tions it had threat­ened.

The biotech’s pro­mo blast this morn­ing sug­gests Affinia is on to a bet­ter AAV tech plat­form than what’s cur­rent­ly in cir­cu­la­tion. Back­ing up that boast are 3 sci­en­tif­ic founders:

Luk Van­den­berghe, Botond Ros­ka and Aaron Tward.

— Van­den­berghe, a pro­fes­sor at Har­vard Med, is cred­it­ed as a co-in­ven­tor of AAV9. He de­vel­oped the tech that was de­vel­oped at the Grous­beck Gene Ther­a­py Cen­ter un­der a col­lab­o­ra­tion be­tween Lon­za and Mass­a­chu­setts Eye and Ear.

— Ros­ka is the di­rec­tor of the In­sti­tute of Mol­e­c­u­lar and Clin­i­cal Oph­thal­mol­o­gy Basel.

— Tward is an as­sis­tant pro­fes­sor at UC-San Fran­cis­co.

Luk Van­den­berghe

“Affinia Ther­a­peu­tics is bring­ing to­geth­er com­ple­men­tary ex­per­tise al­low­ing us to re­al­ize a ra­tio­nal de­sign fu­ture for AAV vec­tors, pro­mot­ers and oth­er com­po­nents of gene ther­a­pies. By lever­ag­ing syn­thet­ic and sys­tems bi­ol­o­gy com­bined with high-through­put screen­ing and tis­sue and sin­gle-cell res­o­lu­tion, we are as­pir­ing to achieve much-need­ed im­proved phar­ma­co­log­i­cal con­trol of this nov­el modal­i­ty in med­i­cine,” said Van­den­berghe in a pre­pared state­ment.

That line­up war­rant­ed a $60 mil­lion launch round from the seed in­vestors at F-Prime Cap­i­tal and New En­ter­prise As­so­ci­ates, with At­las jump­ing in for the first time.

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; 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.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

No­var­tis re­ports two pa­tient deaths af­ter treat­ment with Zol­gens­ma

Two children with spinal muscular atrophy have died after receiving Novartis’ Zolgensma, a gene therapy designed as a one-time treatment for the rare fatal disease.

The deaths, which resulted from acute liver failure, occurred in Russia and Kazakhstan, Novartis confirmed in a statement to Endpoints News. Having notified health authorities across all the markets where Zolgensma is available, it will update the drug label “to specify that fatal acute liver failure has been reported,” a spokesperson wrote.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

House pass­es his­toric drug pric­ing re­forms, lin­ing up decades-in-the-mak­ing win for Biden and De­moc­rats

The US House of Representatives today voted along party lines (all Dems voted for it), 220-207 to pass new, wide-ranging legislation that will allow Medicare drug price negotiations for the first time ever, and cap seniors’ drug expenses to $2,000 per year and seniors’ insulin costs at $35 per month.

Setting up a major victory for President Joe Biden, representatives returned from their summer recess to pass the Inflation Reduction Act, even as many noted the bill would only modestly reduce inflation.

Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

CSL brings Se­qirus, Vi­for un­der par­ent um­brel­la brand in iden­ti­ty re­vamp

CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.