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.”

With just days before President Joe Biden’s Covid-19 booster rollout is set to go into effect, an FDA advisory committee appeared on the verge of not recommending boosters for anyone in the US before a last-minute change of wording laid the groundwork for older adults to have access to a third dose.

The FDA’s adcomm on Vaccines and Related Biological Products (VRBPAC) roundly rejected Pfizer/BioNTech booster shots for all individuals older than 16 by a 16-2 vote Friday afternoon. Soon after, however, the agency posed committee members a new question limiting booster use to the 65-and-older population and individuals at high risk of disease due to occupational exposure or comorbidities.

AstraZeneca and Japanese drugmaker Daiichi Sankyo think they’ve struck gold with their next-gen ADC drug Enhertu, which has shown some striking data in late-stage breast cancer trials and early solid tumor tests. Getting into earlier patients is now the goal, starting with Enhertu’s complete walkover of a Roche drug in second-line breast cancer revealed Saturday.

Enhertu cut the risk of disease progression or death by a whopping 72% (p=<0.0001) compared with Roche’s ADC Kadcyla in second-line unresectable and/or metastatic HER2-positive breast cancer patients who had previously undergone treatment with a Herceptin-chemo combo, according to interim data from the Phase III DESTINY-Breast03 head-to-head study presented at this weekend’s #ESMO21.

In recent months, the battle for PD-(L)1 dominance has spilled over into early cancer with Merck’s Keytruda and Bristol Myers Squibb’s Opdivo all alone on the front lines. Keytruda now has another shell in its bandolier, and it could spell a quick approval.

Keytruda cut the risk of relapse or death by 35% over placebo (p=0.00658) in high-risk, stage 2 melanoma patients who had previously undergone surgery to remove their tumors, according to full data from the Phase III KEYNOTE-716 presented Saturday at #ESMO21.

Merck has worked hard to bring its I/O blockbuster Keytruda into earlier and earlier lines of therapy, and now the wonder drug appears poised to make a quick entry into early advanced cervical cancer.

A combination of Keytruda and chemotherapy with or without Roche’s Avastin cut the risk of death by 33% over chemo with or without Avastin (p=<0.001) in first-line patients with persistent, recurrent or metastatic cervical cancer, according to full data from the Phase III KEYNOTE-826 study presented Saturday at #ESMO21.

Everest Medicines is on a roll with two licensing deals in one week.

The Shanghai-based biotech has paid Sinovent and SinoMab $12 million upfront for the rights to a BTK inhibitor for renal diseases, the company announced Thursday. The deal comes just days after Everest came away with rights to a Covid-19 vaccine in China, Taiwan, Singapore, Thailand and Indonesia.

Everest will pay Sinovent and SinoMab up to $549 million in milestone payments and royalties. The agreement includes tech transfer of Sinovent and SinoMab’s manufacturing process for the candidate, named XNW1011.