Rick Modi, Affinia Therapeutics CEO (Affinia)

Gene ther­a­py play­er Affinia aims to go pub­lic, hop­ing to raise big mon­ey in ef­fort to re­make AAVs

Nine months af­ter Affinia Ther­a­peu­tics raised $110 mil­lion in a Se­ries B, the biotech has set its eyes on the Nas­daq.

The MA-based pre­clin­i­cal gene ther­a­py biotech, which got $60 mil­lion in start­up cap­i­tal from New En­ter­prise As­so­ci­ates, F-Prime and At­las in 2019, filed an S-1 with the SEC on Tues­day, an­nounc­ing ini­tial plans to go pub­lic and pen­cil­ing in a $100 mil­lion raise. Affinia plans to list un­der the tick­er $AF­TX.

Affinia, found­ed back in 2019 by AveX­is vet­er­ans Sean Nolan and Rick Mo­di and an End­points 11 win­ner last year, has been fo­cused on try­ing to solve the lim­i­ta­tions of AAV gene ther­a­py, as high­light­ed by the safe­ty scares and set­backs of the last two years.

Its three sci­en­tif­ic co-founders in­clude Luk Van­den­berghe, who is an as­so­ciate pro­fes­sor at Har­vard Med and di­rec­tor of the Grous­beck Gene Ther­a­py Cen­ter at Mass­a­chu­setts Eye and Ear In­fir­mary in Boston — and is cred­it­ed as a co-in­ven­tor of the more com­mon­ly-used AAV9.

In terms of cur­rent fi­nan­cial stake in the com­pa­ny, NEA man­ag­ing part­ner Ed Math­ers — on Affinia’s board of di­rec­tors — is the largest share­hold­er, own­ing over 11.8 mil­lion shares and equiv­a­lent to 20.5% of the com­pa­ny. Oth­er big share­hold­ers cur­rent­ly in­clude At­las Ven­ture with just shy of 7.5 mil­lion shares, or 13% of Affinia; F-Prime with al­most a 10% stake and 5.7 mil­lion shares; and Lon­za Hous­ton with 4.9 mil­lion shares and an 8.6% stake.

CEO Rick Mo­di has a 1.4% stake with over 800,000 shares, and Van­den­berghe has 1.5 mil­lion shares, or a 2.7% stake.

While the biotech has al­ready raked in over $200 mil­lion in deals and pri­vate fi­nanc­ings, Affinia wants to use what­ev­er mon­ey it rais­es in an IPO to push to­wards clin­i­cal tri­als, pipeline ex­pan­sion and man­u­fac­tur­ing — al­though no spe­cif­ic dol­lar amounts for those ob­jec­tives were list­ed. The mon­ey should fund op­er­a­tions through 2024, it said.

Its lead can­di­date, AF­TX-001, is be­ing de­vel­oped for the treat­ment of metachro­mat­ic leukody­s­tro­phy, a rare ge­net­ic dis­or­der that leads to fat buildup in the body. Its sec­ond can­di­date, AF­TX-002, is in de­vel­op­ment to treat brain metas­tases sec­ondary to HER2+ breast can­cer.

Affinia ex­pects to file INDs on the two can­di­dates in 2023 — the first can­di­date in the first half of the year, and the oth­er can­di­date in the sec­ond half of 2023.

But those aren’t the on­ly things Affinia is work­ing on: They al­so have a sub­stan­tial deal with Ver­tex that Affinia signed in 2020. The S-1 gave fur­ther de­tails on the deal, show­ing it could net Affinia up to $4.7 bil­lion to en­gi­neer cap­sids for use in pro­grams for Duchenne mus­cu­lar dy­s­tro­phy (DMD), my­oton­ic dy­s­tro­phy 1 (DM1) and cys­tic fi­bro­sis (CF).

Alexander Lefterov/Endpoints News

A new can­cer im­munother­a­py brings cau­tious hope for a field long await­ing the next big break­through

Bob Seibert sat silent across from his daughter at their favorite Spanish restaurant near his home in Charleston County, SC, their paella growing cold as he read through all the places in his body doctors found tumors.

He had texted his wife, a pediatric intensive care nurse, when he got the alert that his online chart was ready. Although he saw immediately it was bad, many of the terms — peritoneal, right iliac — were inscrutable. But she was five hours downstate, at a loud group dinner the night before another daughter’s cheer competition.

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Dominic Borie, Kyverna Therapeutics CEO

Well-con­nect­ed, Gilead-backed biotech gets an­oth­er stack of cash to pur­sue CAR-Ts for au­toim­mune dis­ease

Almost exactly two years after its debut at the 2020 JP Morgan confab — and on the heels of a new partnership with the gene editing experts at Intellia — a Gilead-backed, autoimmune disease-focused startup has returned to the well with a clearer outline of just what it plans to do with its CAR-T platform.

Kyverna brought in $85 million in its oversubscribed Series B, the company announced Wednesday. Northpond Ventures led the round, and Westlake Village BioPartners, Vida Ventures, Gilead and Intellia all contributed as well.

Bahija Jallal, Immunocore CEO

BREAK­ING: FDA hur­ries up a quick ap­proval for the world's first TCR -- af­ter a 14-year R&D trek

Over the 14 years since Immunocore was spun out of MediGene in a quest to develop a gamechanging cancer med, the biotech has raised record sums and undergone a major shakeup on a long roller coaster ride of valuations for investors. But they survived and thrived and today they’re popping the champagne corks to celebrate an FDA approval of their first TCR drug.

Immunocore flagged the FDA’s green light for tebentafusp Wednesday morning by highlighting a series of firsts.

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Days af­ter Gilead yanks PI3K drug, In­cyte with­draws NDA for its own PI3K — say­ing con­fir­ma­to­ry tri­als would take too long

The FDA’s intensifying scrutiny on accelerated approvals isn’t just putting pressure on drugmakers with marketed products. It is also subtly reshaping the regulatory dynamics.

Case in point: Incyte announced late Tuesday that it has made the “business decision” to withdraw an NDA for parsaclisib, its oral PI3Kδ inhibitor, after deciding that running the confirmatory studies the agency was asking for to support an accelerated approval wouldn’t be worth it.

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Dan O'Day (Getty Images)

In a set­back, FDA or­ders Gilead to hit the brakes on their late-stage, $5B can­cer play

Gilead’s $5 billion drug magrolimab has run into a serious setback.

The FDA ordered Gilead to halt enrollment on their studies of the drug in combination with azacitidine after investigators reports revealed an “apparent imbalance” in the suspected unexpected serious adverse reactions between study arms. And the halt is raising questions about Gilead’s plans for a quick pitch to regulators.

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Graphic: Alexander Lefterov for Endpoints News

Small biotechs with big drug am­bi­tions threat­en to up­end the tra­di­tion­al drug launch play­book

Of the countless decisions Vlad Coric had to make as Biohaven’s CEO over the past seven years, there was one that felt particularly nerve-wracking: Instead of selling to a Big Pharma, the company decided it would commercialize its migraine drug itself.

“I remember some investors yelling and pounding on the table like, you can’t do this. What are you thinking? You’re going to get crushed by AbbVie,” he recalled.

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Mar­ket­ingRx roundup: Pfiz­er de­buts Pre­vnar 20 TV ads; Lil­ly gets first FDA 2022 pro­mo slap down let­ter

Pfizer debuted its first TV ad for its Prevnar 20 next-generation pneumococcal pneumonia vaccine. In the 60-second spot, several people (actor portrayals) with their ages listed as 65 or older are shown walking into a clinic as they turn to say they’re getting vaccinated with Prevnar 20 because they’re at risk.

The update to Pfizer’s blockbuster Prevnar 13 vaccine was approved in June, and as its name suggests is a vaccine for 20 serotypes — the original 13 plus seven more that cause pneumococcal disease. Pfizer used to spend heavily on TV ads to promote Prevnar 13 in 2018 and 2019 but cut back its TV budgets in the past two fall and winter seasonal spending cycles. Prevnar had been Pfizer’s top-selling drug, notching sales of just under $6 billion in 2020, and was the world’s top-selling vaccine before the Covid-19 vaccines came to market last year.

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Roy Baynes, Merck

FDA bats back Mer­ck’s ‘pipeline in a prod­uct,’ de­mands more ef­fi­ca­cy da­ta

Despite some heavy blowback from analysts, Merck execs maintained an upbeat attitude about the market potential of its chronic cough drug gefapixant. But the confidence may be fading somewhat today as Merck puts out news that the FDA is handing back its application with a CRL.

Dubbed by Merck’s development chief Roy Baynes as a “pipeline in a product” with a variety of potential uses, Merck had fielded positive late-stage data demonstrating the drug’s ability to combat chronic cough. The drug dramatically reduced chronic cough in Phase III, but so did placebo, leaving Merck’s research team with a marginal success on the p-value side of the equation.

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FDA slams door to piv­otal tri­al for bub­ble boy dis­ease gene ther­a­py as Mus­tang Bio runs in­to an­oth­er hold

Mustang Bio is in familiar territory, but that isn’t a place it necessarily wants to be.

The FDA has placed a hold on Mustang Bio’s pivitol trial for its gene therapy to treat patients with bubble boy disease, citing issues surrounding chemistry, manufacturing and controls clearance. It’s the second hold due to CMC issues the company has received in roughly 18 months.

An investigational new drug application was submitted in December 2021. If granted an IND, a Phase II study will then assess safety, tolerability and efficacy of MB-207. If approved by the FDA, the therapy would one day be eligible for a rare pediatric disease voucher.