Af­ter some ear­ly hic­cups in its man­u­fac­tur­ing pro­ce­dures for its gene ther­a­py drug tar­get­ing wet age-re­lat­ed mac­u­lar de­gen­er­a­tion, Cal­i­for­nia biotech Ad­verum has ad­dressed those con­cerns and is prim­ing the pump for a com­mer­cial launch.

The oc­u­lar and oth­er rare dis­ease-fo­cused gene ther­a­py com­pa­ny will in­vest $80 mil­lion in a new cGMP man­u­fac­tur­ing fa­cil­i­ty in North Car­oli­na’s bustling Re­search Tri­an­gle Park that by 2023 will be ready to man­u­fac­ture com­mer­cial sup­ply of AD­VM-022, Ad­verum’s in­trav­it­re­al ther­a­peu­tic, the com­pa­ny said Thurs­day.

The 174,000 square-foot fa­cil­i­ty will in­clude four 1,000-liter biore­ac­tors, with space for fu­ture ex­pan­sion, and will cre­ate more than 200 jobs. In ad­di­tion to its $80 mil­lion in­vest­ment, Ad­verum will gain up to $9 mil­lion in North Car­oli­na state and lo­cal in­cen­tives.

An­gela Thedin­ga

“Our new in-house com­mer­cial man­u­fac­tur­ing fa­cil­i­ty sets the foun­da­tion of our over­all man­u­fac­tur­ing strat­e­gy,” An­gela Thedin­ga, Ad­verum’s chief tech­nol­o­gy of­fi­cer, said in a state­ment. “Our new site will pro­vide ded­i­cat­ed com­mer­cial sup­ply, while we con­tin­ue to lever­age CMO part­ner­ships for flex­i­ble clin­i­cal and ad­di­tion­al com­mer­cial sup­ply.”

In 2020, Phase I tri­al da­ta from AD­VM-022 start­ed to turn a cor­ner af­ter some weak read­outs in the past. In­ves­ti­ga­tors in May re­port­ed that two of three co­horts con­tin­ued to re­spond to treat­ment, with the ma­jor­i­ty still free of the need for res­cue in­jec­tions, while the third co­hort ex­pe­ri­enced few­er side ef­fects than oth­er an­ti-VEGF ther­a­peu­tics.

Lau­rent Fis­ch­er

The year 2019 was more dif­fi­cult for the biotech. In April of that year, the FDA im­posed a clin­i­cal hold on the ap­pli­ca­tion to test AD­VM-022 in hu­mans, ask­ing Ad­verum for more da­ta on the chem­istry, man­u­fac­tur­ing and con­trol process, End­points News re­port­ed. The hold was lift­ed short­ly af­ter.

Then in Sep­tem­ber, Ad­verum re­vealed da­ta that in­di­cat­ed pa­tients with AMD saw their vi­sion de­te­ri­o­rate when treat­ed with AD­VM-022, de­spite signs that the treat­ment was im­prov­ing reti­nal anato­my. Those pa­tients who saw a vi­sion de­te­ri­o­ra­tion lost vi­su­al acu­ity by two let­ters on av­er­age in a 90% con­fi­dence in­ter­val of -9.1 let­ters to +5.1 let­ters, the com­pa­ny re­port­ed from the 24-week OP­TIC tri­al.

In late 2019, the biotech al­so aban­doned de­vel­op­ment of its then-lead ex­per­i­men­tal drug, AD­VM-043, for treat­ing A1AT de­fi­cien­cy.

In a press re­lease an­nounc­ing Ad­verum’s new fa­cil­i­ty, CEO Lau­rent Fis­ch­er was down­right bull­ish on the com­pa­ny’s fu­ture.

“We are build­ing a team with ex­cep­tion­al AAV-gene ther­a­py ex­pe­ri­ence and we are thrilled to join the high­ly-skilled and di­verse North Car­oli­na biotech com­mu­ni­ty,” he said. “This state-of-the-art fa­cil­i­ty will sup­port our com­mer­cial­iza­tion of AD­VM-022, the po­ten­tial first mass mar­ket­ed gene ther­a­py for wet AMD and DME.”

It’s not new for Lonza, the Swiss CDMO nearing its quasquicentennial anniversary, to be in the upper echelon of the biotech manufacturing industry.

But 2020 — as it was for many CDMOs — was a special year even by Lonza’s standards. The company inked a deal to produce 1 billion worldwide doses of Moderna’s Covid-19 vaccine and tapped pharma vet Pierre-Alain Ruffieux to lead its operations, moves which have allowed Lonza to make a myriad of other deals that will continue to ramp up its global production capacity.

More than five years after Corey Fishman and Michael Dunne dusted sulopenem off Pfizer’s shelves — the second castoff antibiotic they’ve brought out of the pharma giant — and founded Iterum Therapeutics around that single drug, they have lined up a quick shot at approval with priority review from the FDA.

The decision, six months from now, will mark a make-or-break moment for a struggling biotech that has just enough cash to keep the lights on until the third quarter.

Immunocore spent much of 2019 dealing with the fallout of the Neil Woodford scandal, as the former star investor’s fall crashed the biotech’s valuation out of unicorn range. Now it turns out that the company spent 2020 dealing with another internal scandal.

The longtime UK biotech darling disclosed in their IPO filing last week that they had fallen victim to an alleged kickback scheme involving one of their employees. After a whistleblower came forward, they said in their F-1, they spent the summer and spring investigating, finding fraud on the part of an employee and two outside vendors.

Longtime Vertex CEO Jeffrey Leiden is taking on a new role.

Leiden has been appointed chairman of Tmunity’s board of directors, the company announced Monday. The move comes about a year and a half after Leiden announced he’d be stepping down from his position at Vertex.

Vertex saw immense growth under Leiden, leading the company from its exit out of hepatitis C, when cures were moving in, and into cystic fibrosis. The company’s cystic fibrosis triple combo therapy Trikafta is already its best-seller, reaching the distinction just six weeks after launch and recording the strongest first quarter of sales for any drug, per some estimates.